메뉴 건너뛰기




Volumn 1, Issue 3, 2001, Pages 294-299

Gene therapy: A strategy for the treatment of inherited muscle diseases?

Author keywords

Gene therapy; Heart; Molecular Medicine; Muscular dystrophy; Myoblast transplantation; Myopathy; Pharmacology; Skeletal muscle; Techniqes Methods; Vectors

Indexed keywords

ANIMAL; GENE THERAPY; GENETICS; HUMAN; MUSCLE DISEASE; PHYSIOLOGY; REVIEW; SKELETAL MUSCLE;

EID: 0035376882     PISSN: 14714892     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1471-4892(01)00052-2     Document Type: Review
Times cited : (2)

References (46)
  • 3
    • 0032926935 scopus 로고    scopus 로고
    • Electropermeabilization of skeletal muscle enhances gene transfer in vivo
    • Mathiesen I. Electropermeabilization of skeletal muscle enhances gene transfer in vivo. Gene Ther. 6:1999;508-514.
    • (1999) Gene Ther , vol.6 , pp. 508-514
    • Mathiesen, I.1
  • 4
    • 0034689634 scopus 로고    scopus 로고
    • Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route
    • 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.
    • 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.
    • (2000) Hum Gene Ther , vol.11 , pp. 701-714
    • 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
  • 5
    • 0033541587 scopus 로고    scopus 로고
    • 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
    • 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.
    • 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.
    • (1999) Hum Gene Ther , vol.10 , pp. 1009-1019
    • Nalbantoglu, J.1    Pari, G.2    Karpati, G.3    Holland, P.C.4
  • 6
    • 0034008961 scopus 로고    scopus 로고
    • The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers
    • 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.
    • 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.
    • (2000) Hum Gene Ther , vol.11 , pp. 521-536
    • 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
  • 8
    • 0035915778 scopus 로고    scopus 로고
    • Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene therapy in the treatment of muscular dystrophies
    • 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.
    • 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.
    • (2001) Hum Gene Ther , vol.12 , pp. 205-215
    • 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
  • 9
    • 0034062095 scopus 로고    scopus 로고
    • Development of new techniques using genetic and tissue engineering for the treatment of severe heart failure
    • 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.
    • (2000) Transplant Proc , vol.32 , pp. 242-244
    • Sawa, Y.1    Fukushima, N.2    Nishimura, M.3    Sakaguchi, T.4    Ohtake, S.5    Matsuda, H.6
  • 10
    • 0033977926 scopus 로고    scopus 로고
    • High-efficiency, long-term cardiac expression of foreign genes in living mouse embryos and neonates
    • 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.
    • (2000) Circulation , vol.101 , pp. 178-184
    • Christensen, G.1    Minamisawa, S.2    Gruber, P.J.3    Wang, Y.4    Chien, K.R.5
  • 11
    • 0028915634 scopus 로고
    • The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants
    • 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.
    • (1995) Gene Ther , vol.2 , pp. 107-115
    • Huard, J.1    Lochmuller, H.2    Acsadi, G.3    Jani, A.4    Massie, B.5    Karpati, G.6
  • 13
    • 0033154485 scopus 로고    scopus 로고
    • Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle
    • 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.
    • 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.
    • (1999) J Gene Med , vol.1 , pp. 280-289
    • 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
  • 14
    • 0033964678 scopus 로고    scopus 로고
    • Developments in gene therapy for muscular dystrophy
    • Hartigan-O'Connor D., Chamberlain J.S. Developments in gene therapy for muscular dystrophy. Microsc Res Tech. 48:2000;223-238.
    • (2000) Microsc Res Tech , vol.48 , pp. 223-238
    • Hartigan-O'Connor, D.1    Chamberlain, J.S.2
  • 15
    • 0034610364 scopus 로고    scopus 로고
    • Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
    • 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.
    • 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.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 13714-13719
    • Wang, B.1    Li, J.2    Xiao, X.3
  • 16
    • 0034611016 scopus 로고    scopus 로고
    • Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion
    • 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.
    • (2000) J Cell Biol , vol.148 , pp. 985-996
    • 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
  • 17
  • 18
    • 0035793047 scopus 로고    scopus 로고
    • Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
    • 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.
    • 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.
    • (2001) Proc Natl Acad Sci USA , vol.98 , pp. 42-47
    • 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
  • 19
    • 0034624974 scopus 로고    scopus 로고
    • Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
    • 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.
    • 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.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 5363-5368
    • Rando, T.A.1    Disatnik, M.H.2    Zhou, L.Z.3
  • 20
    • 0034086776 scopus 로고    scopus 로고
    • In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
    • 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.
    • 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.
    • (2000) Nat Biotechnol , vol.18 , pp. 615-622
    • 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
  • 22
    • 0033544850 scopus 로고    scopus 로고
    • Utrophin lacks the rod domain actin binding activity of dystrophin
    • 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.
    • (1999) J Biol Chem , vol.274 , pp. 35375-35380
    • Amann, K.J.1    Guo, A.W.2    Ervasti, J.M.3
  • 23
    • 0033994291 scopus 로고    scopus 로고
    • Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene
    • 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.
    • (2000) Gene Ther , vol.7 , pp. 201-204
    • Wakefield, P.M.1    Tinsley, J.M.2    Wood, M.J.3    Gilbert, R.4    Karpati, G.5    Davies, K.E.6
  • 24
    • 0033427671 scopus 로고    scopus 로고
    • Nitric oxide and L-arginine cause an accumulation of utrophin at the sarcolemma: A possible compensation for dystrophin loss in Duchenne muscular dystrophy
    • 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.
    • (1999) Neurobiol Dis , vol.6 , pp. 499-507
    • Chaubourt, E.1    Fossier, P.2    Baux, G.3    Leprince, C.4    Israel, M.5    De La Porte, S.6
  • 26
    • 0034631322 scopus 로고    scopus 로고
    • Systemic production of human granulocyte colony-stimulating factor in non-human primates by transplantation of genetically modified myoblasts
    • 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.
    • (2000) Hum Gene Ther , vol.11 , pp. 1277-1288
    • 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
  • 28
    • 0034110642 scopus 로고    scopus 로고
    • A novel means of drug delivery: Myoblast-mediated gene therapy and regulatable retroviral vectors
    • 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.
    • (2000) Annu Rev Pharmacol Toxicol , vol.40 , pp. 295-317
    • Ozawa, C.R.1    Springer, M.L.2    Blau, H.M.3
  • 29
    • 0034111572 scopus 로고    scopus 로고
    • Myoblast transplantation in whole muscle of nonhuman primates
    • This article provides up-to-date methods for efficient myoblast transplantation in primates and good insights for the planning of future clinical protocols.
    • 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.
    • (2000) J Neuropathol Exp Neurol , vol.59 , pp. 197-206
    • Skuk, D.1    Goulet, M.2    Roy, B.3    Tremblay, J.P.4
  • 30
    • 0033950086 scopus 로고    scopus 로고
    • Progress in myoblast transplantation: A potential treatment of dystrophies
    • Skuk D., Tremblay J.P. Progress in myoblast transplantation: a potential treatment of dystrophies. Microsc Res Tech. 48:2000;213-222.
    • (2000) Microsc Res Tech , vol.48 , pp. 213-222
    • Skuk, D.1    Tremblay, J.P.2
  • 31
    • 0034664770 scopus 로고    scopus 로고
    • Pax7 is required for the specification of myogenic satellite cells
    • This paper provides molecular proof that MDSCs and satellite cells (myoblasts) are distinct populations.
    • 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.
    • (2000) Cell , vol.102 , pp. 777-786
    • Seale, P.1    Sabourin, L.A.2    Girgis-Gabardo, A.3    Mansouri, A.4    Gruss, P.5    Rudnicki, M.A.6
  • 33
    • 0035931760 scopus 로고    scopus 로고
    • Intraarterial injection of muscle-derived cd34+sca-1+ stem cells restores dystrophin in mdx mice
    • 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.
    • 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.
    • (2001) J Cell Biol , vol.152 , pp. 335-348
    • 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
  • 34
    • 0032494182 scopus 로고    scopus 로고
    • Development of approaches to improve cell survival in myoblast transfer therapy
    • Qu Z., Balkir L., van Deutekom J.C., Robbins P.D., Pruchnic R., Huard J. Development of approaches to improve cell survival in myoblast transfer therapy. J Cell Biol. 142:1998;1257-1267.
    • (1998) J Cell Biol , vol.142 , pp. 1257-1267
    • Qu, Z.1    Balkir, L.2    Van Deutekom, J.C.3    Robbins, P.D.4    Pruchnic, R.5    Huard, J.6
  • 35
    • 0033594119 scopus 로고    scopus 로고
    • Dynamics of myoblast transplantation reveal a discrete minority of precursors with stem cell-like properties as the myogenic source
    • Beauchamp J.R., Morgan J.E., Pagel C.N., Partridge T.A. Dynamics of myoblast transplantation reveal a discrete minority of precursors with stem cell-like properties as the myogenic source. J Cell Biol. 144:1999;1113-1122.
    • (1999) J Cell Biol , vol.144 , pp. 1113-1122
    • Beauchamp, J.R.1    Morgan, J.E.2    Pagel, C.N.3    Partridge, T.A.4
  • 36
    • 0033598374 scopus 로고    scopus 로고
    • Dystrophin expression in the mdx mouse restored by stem cell transplantation
    • This article reports the first experiments on MDSCs, from their purification, their characterisation and their intravenous injection in mdx mice.
    • Gussoni E., Soneoka Y., Strickland C.D., Buzney E.A., Khan M.K., Flint A.F., Kunkel L.M., Mulligan R.C. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature. 401:1999;390-394. This article reports the first experiments on MDSCs, from their purification, their characterisation and their intravenous injection in mdx mice.
    • (1999) Nature , vol.401 , pp. 390-394
    • Gussoni, E.1    Soneoka, Y.2    Strickland, C.D.3    Buzney, E.A.4    Khan, M.K.5    Flint, A.F.6    Kunkel, L.M.7    Mulligan, R.C.8
  • 37
    • 0033945869 scopus 로고    scopus 로고
    • Evidence for a myogenic stem cell that is exhausted in dystrophic muscle
    • Many of the studies on stem cells use normal donor animals. To be useful for autologous cell transplantation, however, it has to be assessed that MDSCs possess the same properties in dystrophic and normal animals. This article shows important differences in the MDSCs from the mdx mouse that could limit their use in ex vivo gene therapy.
    • Heslop L., Morgan J.E., Partridge T.A. Evidence for a myogenic stem cell that is exhausted in dystrophic muscle. J Cell Sci. 113:2000;2299-2308. Many of the studies on stem cells use normal donor animals. To be useful for autologous cell transplantation, however, it has to be assessed that MDSCs possess the same properties in dystrophic and normal animals. This article shows important differences in the MDSCs from the mdx mouse that could limit their use in ex vivo gene therapy.
    • (2000) J Cell Sci , vol.113 , pp. 2299-2308
    • Heslop, L.1    Morgan, J.E.2    Partridge, T.A.3
  • 41
    • 0033958294 scopus 로고    scopus 로고
    • Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy
    • Xiao X., Li J., Tsao Y.P., Dressman D., Hoffman E.P., Watchko J.F. Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy. J Virol. 74:2000;1436-1442.
    • (2000) J Virol , vol.74 , pp. 1436-1442
    • Xiao, X.1    Li, J.2    Tsao, Y.P.3    Dressman, D.4    Hoffman, E.P.5    Watchko, J.F.6
  • 42
    • 0034689207 scopus 로고    scopus 로고
    • Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors
    • Gene therapy has been promising for the treatment of myopathies; however, few of the published experimental protocols have the potential to lead to actual clinical trials. This phase I trial, therefore, sets an important milestone for the evaluation of safety and feasibility of such an approach.
    • Stedman H., Wilson J.M., Finke R., Kleckner A.L., Mendell J. Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors. Hum Gene Ther. 11:2000;777-790. Gene therapy has been promising for the treatment of myopathies; however, few of the published experimental protocols have the potential to lead to actual clinical trials. This phase I trial, therefore, sets an important milestone for the evaluation of safety and feasibility of such an approach.
    • (2000) Hum Gene Ther , vol.11 , pp. 777-790
    • Stedman, H.1    Wilson, J.M.2    Finke, R.3    Kleckner, A.L.4    Mendell, J.5
  • 45
    • 0033529902 scopus 로고    scopus 로고
    • Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase
    • 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.
    • 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.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 8861-8866
    • Amalfitano, A.1    McVie-Wylie, A.J.2    Hu, H.3    Dawson, T.L.4    Raben, N.5    Plotz, P.6    Chen, Y.T.7
  • 46
    • 0034212568 scopus 로고    scopus 로고
    • Towards a molecular therapy for glycogen storage disease type II (Pompe disease)
    • Chen Y.T., Amalfitano A. Towards a molecular therapy for glycogen storage disease type II (Pompe disease). Mol Med Today. 6:2000;245-251.
    • (2000) Mol Med Today , vol.6 , pp. 245-251
    • Chen, Y.T.1    Amalfitano, A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.