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1
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0034724857
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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
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Cavazzana-Calvo M., Hacein-Bey S., de Saint Basile G., Gross F., Yvon E., Nusbaum P., Selz F., Hue C., Certain S., Casanova J.L., et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288:2000;669-672.
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(2000)
Science
, vol.288
, pp. 669-672
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Cavazzana-Calvo, M.1
Hacein-Bey, S.2
De Saint Basile, G.3
Gross, F.4
Yvon, E.5
Nusbaum, P.6
Selz, F.7
Hue, C.8
Certain, S.9
Casanova, J.L.10
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2
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0033773060
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Functional EGFP-dystrophin fusion proteins for gene therapy vector development
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Chapdelaine P., Moisset P.A., Campeau P., Asselin I., Vilquin J.T., Tremblay J.P. Functional EGFP-dystrophin fusion proteins for gene therapy vector development. Protein Eng. 13:2000;611-615.
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(2000)
Protein Eng
, vol.13
, pp. 611-615
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Chapdelaine, P.1
Moisset, P.A.2
Campeau, P.3
Asselin, I.4
Vilquin, J.T.5
Tremblay, J.P.6
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3
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0032926935
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Electropermeabilization of skeletal muscle enhances gene transfer in vivo
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Mathiesen I. Electropermeabilization of skeletal muscle enhances gene transfer in vivo. Gene Ther. 6:1999;508-514.
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(1999)
Gene Ther
, vol.6
, pp. 508-514
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Mathiesen, I.1
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4
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0034689634
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Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route
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The elaboration of an effective, systemic delivery method is imperative to treat myopathies. This article describes new ways to help adenoviruses cross the endothelial barrier after intra-arterial injection of the vector.
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Cho W.K., Ebihara S., Nalbantoglu J., Gilbert R., Massie B., Holland P., Karpati G., Petrof B.J. Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route. Hum Gene Ther. 11:2000;701-714. The elaboration of an effective, systemic delivery method is imperative to treat myopathies. This article describes new ways to help adenoviruses cross the endothelial barrier after intra-arterial injection of the vector.
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(2000)
Hum Gene Ther
, vol.11
, pp. 701-714
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Cho, W.K.1
Ebihara, S.2
Nalbantoglu, J.3
Gilbert, R.4
Massie, B.5
Holland, P.6
Karpati, G.7
Petrof, B.J.8
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5
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0033541587
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Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells
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Adenoviral vectors transduce neonate muscles very efficiently; however, adult muscle is much harder to transduce. These authors hypothesize that the downregulation of the primary adenoviral receptor, which is observed during the maturation of the muscle, could be the cause of this problem.
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Nalbantoglu J., Pari G., Karpati G., Holland P.C. Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells. Hum Gene Ther. 10:1999;1009-1019. Adenoviral vectors transduce neonate muscles very efficiently; however, adult muscle is much harder to transduce. These authors hypothesize that the downregulation of the primary adenoviral receptor, which is observed during the maturation of the muscle, could be the cause of this problem.
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(1999)
Hum Gene Ther
, vol.10
, pp. 1009-1019
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Nalbantoglu, J.1
Pari, G.2
Karpati, G.3
Holland, P.C.4
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6
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0034008961
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The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers
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Adenoviral transduction of adult muscle fibers is not efficient. Whether this is because of the downregulation of the receptors and/or changes in the extracellular matrix, the authors show that AAV transduction is much less affected by these changes.
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Pruchnic R., Cao B., Peterson Z.Q., Xiao X., Li J., Samulski R.J., Epperly M., Huard J. The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers. Hum Gene Ther. 11:2000;521-536. Adenoviral transduction of adult muscle fibers is not efficient. Whether this is because of the downregulation of the receptors and/or changes in the extracellular matrix, the authors show that AAV transduction is much less affected by these changes.
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(2000)
Hum Gene Ther
, vol.11
, pp. 521-536
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Pruchnic, R.1
Cao, B.2
Peterson, Z.Q.3
Xiao, X.4
Li, J.5
Samulski, R.J.6
Epperly, M.7
Huard, J.8
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7
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0032907636
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Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector
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Greelish J.P., Su L.T., Lankford E.B., Burkman J.M., Chen H., Konig S.K., Mercier I.M., Desjardins P.R., Mitchell M.A., Zheng X.G., et al. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med. 5:1999;439-443.
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(1999)
Nat Med
, vol.5
, pp. 439-443
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Greelish, J.P.1
Su, L.T.2
Lankford, E.B.3
Burkman, J.M.4
Chen, H.5
Konig, S.K.6
Mercier, I.M.7
Desjardins, P.R.8
Mitchell, M.A.9
Zheng, X.G.10
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8
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0035915778
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Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene therapy in the treatment of muscular dystrophies
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The authors show the importance of the choice of promoter for the treatment of muscle disease. Expression of an immunogenic transgene in gamma-sarcoglycan-deficient mice was observed only when the muscle-specific creatine kinase promoter was used.
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Cordier L., Gao G., Hack A.A., McNally E.M., Wilson J.M., Chirmule N., Sweeney H.L. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene therapy in the treatment of muscular dystrophies. Hum Gene Ther. 12:2001;205-215. The authors show the importance of the choice of promoter for the treatment of muscle disease. Expression of an immunogenic transgene in gamma-sarcoglycan-deficient mice was observed only when the muscle-specific creatine kinase promoter was used.
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(2001)
Hum Gene Ther
, vol.12
, pp. 205-215
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Cordier, L.1
Gao, G.2
Hack, A.A.3
McNally, E.M.4
Wilson, J.M.5
Chirmule, N.6
Sweeney, H.L.7
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9
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0034062095
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Development of new techniques using genetic and tissue engineering for the treatment of severe heart failure
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Sawa Y., Fukushima N., Nishimura M., Sakaguchi T., Ohtake S., Matsuda H. Development of new techniques using genetic and tissue engineering for the treatment of severe heart failure. Transplant Proc. 32:2000;242-244.
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(2000)
Transplant Proc
, vol.32
, pp. 242-244
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Sawa, Y.1
Fukushima, N.2
Nishimura, M.3
Sakaguchi, T.4
Ohtake, S.5
Matsuda, H.6
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10
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0033977926
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High-efficiency, long-term cardiac expression of foreign genes in living mouse embryos and neonates
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Christensen G., Minamisawa S., Gruber P.J., Wang Y., Chien K.R. High-efficiency, long-term cardiac expression of foreign genes in living mouse embryos and neonates. Circulation. 101:2000;178-184.
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(2000)
Circulation
, vol.101
, pp. 178-184
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Christensen, G.1
Minamisawa, S.2
Gruber, P.J.3
Wang, Y.4
Chien, K.R.5
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11
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0028915634
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The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants
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Huard J., Lochmuller H., Acsadi G., Jani A., Massie B., Karpati G. The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther. 2:1995;107-115.
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(1995)
Gene Ther
, vol.2
, pp. 107-115
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Huard, J.1
Lochmuller, H.2
Acsadi, G.3
Jani, A.4
Massie, B.5
Karpati, G.6
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12
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0032128353
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Efficient gene transfer into cardiac myocytes using adeno-associated virus (AAV) vectors
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Maeda Y., Ikeda U., Shimpo M., Ueno S., Ogasawara Y., Urabe M., Kume A., Takizawa T., Saito T., Colosi P., et al. Efficient gene transfer into cardiac myocytes using adeno-associated virus (AAV) vectors. J Mol Cell Cardiol. 30:1998;1341-1348.
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(1998)
J Mol Cell Cardiol
, vol.30
, pp. 1341-1348
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Maeda, Y.1
Ikeda, U.2
Shimpo, M.3
Ueno, S.4
Ogasawara, Y.5
Urabe, M.6
Kume, A.7
Takizawa, T.8
Saito, T.9
Colosi, P.10
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13
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0033154485
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Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle
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Herpes viruses are one of the few potential candidates for the delivery of full-length dystrophin because of their large load capacity (up to 135 kb). In this article, the authors show that these vectors can perform efficient transduction of full-length dystrophin by direct intramuscular injection or by an ex vivo approach.
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Akkaraju G.R., Huard J., Hoffman E.P., Goins W.F., Pruchnic R., Watkins S.C., Cohen J.B., Glorioso J.C. Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle. J Gene Med. 1:1999;280-289. Herpes viruses are one of the few potential candidates for the delivery of full-length dystrophin because of their large load capacity (up to 135 kb). In this article, the authors show that these vectors can perform efficient transduction of full-length dystrophin by direct intramuscular injection or by an ex vivo approach.
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(1999)
J Gene Med
, vol.1
, pp. 280-289
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Akkaraju, G.R.1
Huard, J.2
Hoffman, E.P.3
Goins, W.F.4
Pruchnic, R.5
Watkins, S.C.6
Cohen, J.B.7
Glorioso, J.C.8
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15
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0034610364
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Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
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In this paper, the authors describe a new dystrophin mini-gene that is small enough to be packaged in AAV vectors, thus facilitating its delivery, and that can completely prevent the development of the dystrophic phenotype when injected into young rat pups. Moreover, this transgene has been shown to efficiently protect muscle fibers from high mechanical stress. These features make this new transgene very attractive for future DMD gene therapy trials.
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Wang B., Li J., Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci USA. 97:2000;13714-13719. In this paper, the authors describe a new dystrophin mini-gene that is small enough to be packaged in AAV vectors, thus facilitating its delivery, and that can completely prevent the development of the dystrophic phenotype when injected into young rat pups. Moreover, this transgene has been shown to efficiently protect muscle fibers from high mechanical stress. These features make this new transgene very attractive for future DMD gene therapy trials.
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(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 13714-13719
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Wang, B.1
Li, J.2
Xiao, X.3
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16
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0034611016
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Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion
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Lu Q.L., Morris G.E., Wilton S.D., Ly T., Artem'yeva O.V., Strong P., Partridge T.A. Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion. J Cell Biol. 148:2000;985-996.
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(2000)
J Cell Biol
, vol.148
, pp. 985-996
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Lu, Q.L.1
Morris, G.E.2
Wilton, S.D.3
Ly, T.4
Artem'Yeva, O.V.5
Strong, P.6
Partridge, T.A.7
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17
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0033044501
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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides
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Wilton S.D., Lloyd F., Carville K., Fletcher S., Honeyman K., Agrawal S., Kole R. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. Neuromuscul Disord. 9:1999;330-338.
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(1999)
Neuromuscul Disord
, vol.9
, pp. 330-338
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Wilton, S.D.1
Lloyd, F.2
Carville, K.3
Fletcher, S.4
Honeyman, K.5
Agrawal, S.6
Kole, R.7
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18
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0035793047
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Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
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The authors present an interesting alternative to 'standard' gene therapy. By modifying the splicing of the mutated dystrophin RNA with antisense oligoribonucleotides, they have successfully produced 'revertant' muscle fibers expressing truncated versions of dystrophin in mdx mice.
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Mann C.J., Honeyman K., Cheng A.J., Ly T., Lloyd F., Fletcher S., Morgan J.E., Partridge T.A., Wilton S.D. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA. 98:2001;42-47. The authors present an interesting alternative to 'standard' gene therapy. By modifying the splicing of the mutated dystrophin RNA with antisense oligoribonucleotides, they have successfully produced 'revertant' muscle fibers expressing truncated versions of dystrophin in mdx mice.
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(2001)
Proc Natl Acad Sci USA
, vol.98
, pp. 42-47
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Mann, C.J.1
Honeyman, K.2
Cheng, A.J.3
Ly, T.4
Lloyd, F.5
Fletcher, S.6
Morgan, J.E.7
Partridge, T.A.8
Wilton, S.D.9
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19
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0034624974
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Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
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The in situ correction of a mutated gene is the best conceivable therapy for evident reasons. The authors show in this article that such a feat is conceivable, even if the in vivo results were too low to be clinically relevant.
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Rando T.A., Disatnik M.H., Zhou L.Z. Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proc Natl Acad Sci USA. 97:2000;5363-5368. The in situ correction of a mutated gene is the best conceivable therapy for evident reasons. The authors show in this article that such a feat is conceivable, even if the in vivo results were too low to be clinically relevant.
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(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 5363-5368
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Rando, T.A.1
Disatnik, M.H.2
Zhou, L.Z.3
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20
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0034086776
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In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
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Dystrophic (mdx) mice are most commonly used as models for DMD; however, it would be preferable to use large animal models to fully assess the feasibility of a gene therapy protocol. This article describes how gene correction experiments in mdx mice have been reproduced in the dystrophic dog.
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Bartlett R.J., Stockinger S., Denis M.M., Bartlett W.T., Inverardi L., Le T.T., Man N., Morris G.E., Bogan D.J., Metcalf-Bogan J., et al. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide. Nat Biotechnol. 18:2000;615-622. Dystrophic (mdx) mice are most commonly used as models for DMD; however, it would be preferable to use large animal models to fully assess the feasibility of a gene therapy protocol. This article describes how gene correction experiments in mdx mice have been reproduced in the dystrophic dog.
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(2000)
Nat Biotechnol
, vol.18
, pp. 615-622
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Bartlett, R.J.1
Stockinger, S.2
Denis, M.M.3
Bartlett, W.T.4
Inverardi, L.5
Le, T.T.6
Man, N.7
Morris, G.E.8
Bogan, D.J.9
Metcalf-Bogan, J.10
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21
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0034622814
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Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice
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Ebihara S., Guibinga G.H., Gilbert R., Nalbantoglu J., Massie B., Karpati G., Petrof B.J. Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice. Physiol Genomics. 3:2000;133-144.
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(2000)
Physiol Genomics
, vol.3
, pp. 133-144
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Ebihara, S.1
Guibinga, G.H.2
Gilbert, R.3
Nalbantoglu, J.4
Massie, B.5
Karpati, G.6
Petrof, B.J.7
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22
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0033544850
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Utrophin lacks the rod domain actin binding activity of dystrophin
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Amann K.J., Guo A.W., Ervasti J.M. Utrophin lacks the rod domain actin binding activity of dystrophin. J Biol Chem. 274:1999;35375-35380.
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(1999)
J Biol Chem
, vol.274
, pp. 35375-35380
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Amann, K.J.1
Guo, A.W.2
Ervasti, J.M.3
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23
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0033994291
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Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene
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Wakefield P.M., Tinsley J.M., Wood M.J., Gilbert R., Karpati G., Davies K.E. Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene. Gene Ther. 7:2000;201-204.
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(2000)
Gene Ther
, vol.7
, pp. 201-204
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Wakefield, P.M.1
Tinsley, J.M.2
Wood, M.J.3
Gilbert, R.4
Karpati, G.5
Davies, K.E.6
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24
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0033427671
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Nitric oxide and L-arginine cause an accumulation of utrophin at the sarcolemma: A possible compensation for dystrophin loss in Duchenne muscular dystrophy
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Chaubourt E., Fossier P., Baux G., Leprince C., Israel M., De La Porte S. Nitric oxide and L-arginine cause an accumulation of utrophin at the sarcolemma: a possible compensation for dystrophin loss in Duchenne muscular dystrophy. Neurobiol Dis. 6:1999;499-507.
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(1999)
Neurobiol Dis
, vol.6
, pp. 499-507
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Chaubourt, E.1
Fossier, P.2
Baux, G.3
Leprince, C.4
Israel, M.5
De La Porte, S.6
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25
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0033843263
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Recombinant adenoviral vectors have adjuvant activity and stimulate T cell responses against tumor cells
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Geutskens S.B., van der Eb M.M., Plomp A.C., Jonges L.E., Cramer S.J., Ensink N.G., Kuppen P.J., Hoeben R.C. Recombinant adenoviral vectors have adjuvant activity and stimulate T cell responses against tumor cells. Gene Ther. 7:2000;1410-1416.
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(2000)
Gene Ther
, vol.7
, pp. 1410-1416
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Geutskens, S.B.1
Van Der Eb, M.M.2
Plomp, A.C.3
Jonges, L.E.4
Cramer, S.J.5
Ensink, N.G.6
Kuppen, P.J.7
Hoeben, R.C.8
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26
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0034631322
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Systemic production of human granulocyte colony-stimulating factor in non-human primates by transplantation of genetically modified myoblasts
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Moisset P.A., Bonham L., Skuk D., Koeberl D., Brussee V., Goulet M., Roy B., Asselin I., Miller A.D., Tremblay J.P. Systemic production of human granulocyte colony-stimulating factor in non-human primates by transplantation of genetically modified myoblasts. Hum Gene Ther. 11:2000;1277-1288.
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(2000)
Hum Gene Ther
, vol.11
, pp. 1277-1288
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Moisset, P.A.1
Bonham, L.2
Skuk, D.3
Koeberl, D.4
Brussee, V.5
Goulet, M.6
Roy, B.7
Asselin, I.8
Miller, A.D.9
Tremblay, J.P.10
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27
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0033104799
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Tissue-engineered human bioartificial muscles expressing a foreign recombinant protein for gene therapy
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Powell C., Shansky J., Del Tatto M., Forman D.E., Hennessey J., Sullivan K., Zielinski B.A., Vandenburgh H.H. Tissue-engineered human bioartificial muscles expressing a foreign recombinant protein for gene therapy. Hum Gene Ther. 10:1999;565-577.
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(1999)
Hum Gene Ther
, vol.10
, pp. 565-577
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Powell, C.1
Shansky, J.2
Del Tatto, M.3
Forman, D.E.4
Hennessey, J.5
Sullivan, K.6
Zielinski, B.A.7
Vandenburgh, H.H.8
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28
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0034110642
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A novel means of drug delivery: Myoblast-mediated gene therapy and regulatable retroviral vectors
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Ozawa C.R., Springer M.L., Blau H.M. A novel means of drug delivery: myoblast-mediated gene therapy and regulatable retroviral vectors. Annu Rev Pharmacol Toxicol. 40:2000;295-317.
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(2000)
Annu Rev Pharmacol Toxicol
, vol.40
, pp. 295-317
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Ozawa, C.R.1
Springer, M.L.2
Blau, H.M.3
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29
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0034111572
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Myoblast transplantation in whole muscle of nonhuman primates
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This article provides up-to-date methods for efficient myoblast transplantation in primates and good insights for the planning of future clinical protocols.
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Skuk D., Goulet M., Roy B., Tremblay J.P. Myoblast transplantation in whole muscle of nonhuman primates. J Neuropathol Exp Neurol. 59:2000;197-206. This article provides up-to-date methods for efficient myoblast transplantation in primates and good insights for the planning of future clinical protocols.
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(2000)
J Neuropathol Exp Neurol
, vol.59
, pp. 197-206
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Skuk, D.1
Goulet, M.2
Roy, B.3
Tremblay, J.P.4
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30
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0033950086
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Progress in myoblast transplantation: A potential treatment of dystrophies
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Skuk D., Tremblay J.P. Progress in myoblast transplantation: a potential treatment of dystrophies. Microsc Res Tech. 48:2000;213-222.
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(2000)
Microsc Res Tech
, vol.48
, pp. 213-222
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Skuk, D.1
Tremblay, J.P.2
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31
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0034664770
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Pax7 is required for the specification of myogenic satellite cells
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This paper provides molecular proof that MDSCs and satellite cells (myoblasts) are distinct populations.
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Seale P., Sabourin L.A., Girgis-Gabardo A., Mansouri A., Gruss P., Rudnicki M.A. Pax7 is required for the specification of myogenic satellite cells. Cell. 102:2000;777-786. This paper provides molecular proof that MDSCs and satellite cells (myoblasts) are distinct populations.
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(2000)
Cell
, vol.102
, pp. 777-786
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Seale, P.1
Sabourin, L.A.2
Girgis-Gabardo, A.3
Mansouri, A.4
Gruss, P.5
Rudnicki, M.A.6
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32
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0034605069
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Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing
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Lee J.Y., Qu-Petersen Z., Cao B., Kimura S., Jankowski R., Cummins J., Usas A., Gates C., Robbins P., Wernig A., et al. Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing. J Cell Biol. 150:2000;1085-1100.
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(2000)
J Cell Biol
, vol.150
, pp. 1085-1100
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Lee, J.Y.1
Qu-Petersen, Z.2
Cao, B.3
Kimura, S.4
Jankowski, R.5
Cummins, J.6
Usas, A.7
Gates, C.8
Robbins, P.9
Wernig, A.10
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33
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0035931760
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Intraarterial injection of muscle-derived cd34+sca-1+ stem cells restores dystrophin in mdx mice
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This article confirms that MDSCs migrate and attach to blood vessels following intra-arterial delivery. They then migrate in the muscles where they can fuse with existing muscle fibers. The fusion with muscle fibers is, however, increased when both the blood vessels and the muscle fibers are damaged a few days after the intra-arterial delivery.
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Torrente Y., Tremblay J.P., Pisati F., Belicchi M., Rossi B., Sironi M., Fortunato F., el Fahime M., D'angelo M.G., Caron N.J., et al. Intraarterial injection of muscle-derived cd34+sca-1+ stem cells restores dystrophin in mdx mice. J Cell Biol. 152:2001;335-348. This article confirms that MDSCs migrate and attach to blood vessels following intra-arterial delivery. They then migrate in the muscles where they can fuse with existing muscle fibers. The fusion with muscle fibers is, however, increased when both the blood vessels and the muscle fibers are damaged a few days after the intra-arterial delivery.
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(2001)
J Cell Biol
, vol.152
, pp. 335-348
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Torrente, Y.1
Tremblay, J.P.2
Pisati, F.3
Belicchi, M.4
Rossi, B.5
Sironi, M.6
Fortunato, F.7
El Fahime, M.8
D'Angelo, M.G.9
Caron, N.J.10
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34
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0032494182
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44
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Intramuscular delivery of viral vectors has only led to local correction of glycogen storage disease. This paper reports the systemic correction of the disease by a single injection of a second-generation adenoviral vector. Although the expression of human GAA was shown to decrease rapidly after the injection, the experiment emphasises the importance of delivery route and choice of vector for this type of disease.
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Amalfitano A., McVie-Wylie A.J., Hu H., Dawson T.L., Raben N., Plotz P., Chen Y.T. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase. Proc Natl Acad Sci USA. 96:1999;8861-8866. Intramuscular delivery of viral vectors has only led to local correction of glycogen storage disease. This paper reports the systemic correction of the disease by a single injection of a second-generation adenoviral vector. Although the expression of human GAA was shown to decrease rapidly after the injection, the experiment emphasises the importance of delivery route and choice of vector for this type of disease.
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