-
1
-
-
84879867061
-
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
-
Aiuti, A., et al. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341, 1233151.
-
(2013)
Science
, vol.341
, pp. 1233151
-
-
Aiuti, A.1
-
2
-
-
84879873039
-
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
-
Biffi, A., et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158.
-
(2013)
Science
, vol.341
, pp. 1233158
-
-
Biffi, A.1
-
3
-
-
10744224015
-
LEKTI proteolytic processing in human primary keratinocytes, tissue distribution and defective expression in Netherton syndrome
-
DOI 10.1093/hmg/ddg247
-
Bitoun, E., et al. (2003). LEKTI proteolytic processing in human primary keratinocytes, tissue distribution and defective expression in Netherton syndrome. Hum. Mol. Genet. 12, 2417-2430. (Pubitemid 37220407)
-
(2003)
Human Molecular Genetics
, vol.12
, Issue.19
, pp. 2417-2430
-
-
Bitoun, E.1
Micheloni, A.2
Lamant, L.3
Bonnart, C.4
Tartaglia-Polcini, A.5
Cobbold, C.6
Al Saati, T.7
Mariotti, F.8
Mazareeuw-Hautier, J.9
Boralevi, F.10
Hohl, D.11
Harper, J.12
Bodemer, C.13
D'Alessio, M.14
Hovnanian, A.15
-
4
-
-
77949752021
-
Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Neth-erton syndrome through filaggrin and lipid misprocessing
-
Bonnart, C., et al. (2010). Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Neth-erton syndrome through filaggrin and lipid misprocessing. J. Clin. Invest. 120, 871-882.
-
(2010)
J. Clin. Invest.
, vol.120
, pp. 871-882
-
-
Bonnart, C.1
-
5
-
-
66049125101
-
Kallikrein 5 induces atopic dermatitis-like lesions through PAR2-mediated thymic stromal lymphopoie-tin expression in Netherton syndrome
-
Briot, A., et al. (2009). Kallikrein 5 induces atopic dermatitis-like lesions through PAR2-mediated thymic stromal lymphopoie-tin expression in Netherton syndrome. J. Exp. Med. 206, 1135-1147.
-
(2009)
J. Exp. Med.
, vol.206
, pp. 1135-1147
-
-
Briot, A.1
-
6
-
-
84866652391
-
Gene therapy of skin adhesion disorders (mini review)
-
Cavazza, A., and Mavilio, F. (2012). Gene therapy of skin adhesion disorders (mini review). Curr. Pharm. Biotechnol. 13, 1868-1876.
-
(2012)
Curr. Pharm. Biotechnol.
, vol.13
, pp. 1868-1876
-
-
Cavazza, A.1
Mavilio, F.2
-
7
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
-
Cavazzana-Calvo, M., et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322.
-
(2010)
Nature
, vol.467
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
-
8
-
-
0034120666
-
Mutations in SPINK5, encoding a serine protease inhibitor, cause Netherton syndrome
-
DOI 10.1038/75977
-
Chavanas, S., et al. (2000). Mutations in SPINK5, encoding a serine protease inhibitor, cause Netherton syndrome. Nat. Genet. 25, 141-142. (Pubitemid 30394980)
-
(2000)
Nature Genetics
, vol.25
, Issue.2
, pp. 141-142
-
-
Chavanas, S.1
Bodemer, C.2
Rochat, A.3
Hamel-Teillac, D.4
Ali, M.5
Irvine, A.D.6
Bonafe, J.-L.7
Wilkinson, J.8
Taieb, A.9
Barrandon, Y.10
Harper, J.I.11
De Prost, Y.12
Hovnanian, A.13
-
9
-
-
0029906097
-
Corrective gene transfer in the human skin disorder lamellar ichthyosis
-
DOI 10.1038/nm1196-1263
-
Choate, K.A., Medalie, D.A., Morgan, J.R., and Khavari, P.A. (1996). Corrective gene transfer in the human skin disorder lamellar ichthyosis. Nat. Med. 2, 1263-1267. (Pubitemid 26375415)
-
(1996)
Nature Medicine
, vol.2
, Issue.11
, pp. 1263-1267
-
-
Choate, K.A.1
Medalie, D.A.2
Morgan, J.R.3
Khavari, P.A.4
-
10
-
-
34548515280
-
LEKTI fragments specifically inhibit KLK5, KLK7, and KLK14 and control desquamation through a pH-dependent interaction
-
DOI 10.1091/mbc.E07-02-0124
-
Deraison, C., et al. (2007). LEKTI fragments specifically inhibit KLK5, KLK7, and KLK14 and control desquamation through a pH-dependent interaction. Mol. Biol. Cell 18, 3607-3619. (Pubitemid 47378698)
-
(2007)
Molecular Biology of the Cell
, vol.18
, Issue.9
, pp. 3607-3619
-
-
Deraison, C.1
Bonnart, C.2
Lopez, F.3
Besson, C.4
Robinson, R.5
Jayakumar, A.6
Wagberg, F.7
Brattsand, M.8
Hachem, J.P.9
Leonardsson, G.10
Hovnanian, A.11
-
11
-
-
85028120068
-
Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts
-
Di, W.L., et al. (2011). Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts. Mol. Ther. 19, 408-416.
-
(2011)
Mol. Ther.
, vol.19
, pp. 408-416
-
-
Di, W.L.1
-
12
-
-
84855867684
-
Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression
-
Di, W.L., et al. (2012). Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression. Hum. Gene Ther. 23, 83-90.
-
(2012)
Hum. Gene Ther.
, vol.23
, pp. 83-90
-
-
Di, W.L.1
-
13
-
-
28844473475
-
HK5 and hK7, two serine proteinases abundant in human skin, are inhibited by LEKTI domain 6
-
DOI 10.1111/j.1365-2133.2005.06834.x
-
Egelrud, T., et al. (2005). hK5 and hK7, two serine proteinases abundant in human skin, are inhibited by LEKTI domain 6. Br. J. Dermatol. 153, 1200-1203. (Pubitemid 41779577)
-
(2005)
British Journal of Dermatology
, vol.153
, Issue.6
, pp. 1200-1203
-
-
Egelrud, T.1
Brattsand, M.2
Kreutzmann, P.3
Walden, M.4
Vitzithum, K.5
Marx, U.C.6
Forssmann, W.G.7
Magert, H.-J.8
-
14
-
-
0029918009
-
Loss of expression of a retrovirus-transduced gene in human keratinocytes
-
Fenjves, E.S., Yao, S.N., Kurachi, K., and Taichman, L.B. (1996). Loss of expression of a retrovirus-transduced gene in human keratinocytes. J. Invest. Dermatol. 106, 576-578.
-
(1996)
J. Invest. Dermatol.
, vol.106
, pp. 576-578
-
-
Fenjves, E.S.1
Yao, S.N.2
Kurachi, K.3
Taichman, L.B.4
-
15
-
-
25144508631
-
Assessment of optimal transduction of primary human skin keratinocytes by viral vectors
-
DOI 10.1002/jgm.768
-
Gagnoux-Palacios, L., et al. (2005). Assessment of optimal transduction of primary human skin keratinocytes by viral vectors. J. Gene Med. 7, 1178-1186. (Pubitemid 41335170)
-
(2005)
Journal of Gene Medicine
, vol.7
, Issue.9
, pp. 1178-1186
-
-
Gagnoux-Palacios, L.1
Hervouet, C.2
Spirito, F.3
Roques, S.4
Mezzina, M.5
Danos, O.6
Meneguzzi, G.7
-
16
-
-
0027465539
-
Towards gene therapy for haemophilia B using primary human keratinocytes
-
DOI 10.1038/ng0293-180
-
Gerrard, A.J., Hudson, D.L., Brownlee, G.G., and Watt, F.M. (1993). Towards gene therapy for haemophilia B using primary human keratinocytes. Nat. Genet. 3, 180-183. (Pubitemid 23079803)
-
(1993)
Nature Genetics
, vol.3
, Issue.2
, pp. 180-183
-
-
Gerrard, A.J.1
Hudson, D.L.2
Brownlee, G.G.3
Watt, F.M.4
-
17
-
-
0036790465
-
Durable and stratum-specific gene expression in epidermis
-
Ghazizadeh, S., Doumeng, C., and Taichman, L.B. (2002). Durable and stratum-specific gene expression in epidermis. Gene Ther. 9, 1278-1285.
-
(2002)
Gene Ther.
, vol.9
, pp. 1278-1285
-
-
Ghazizadeh, S.1
Doumeng, C.2
Taichman, L.B.3
-
18
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
DOI 10.1126/science.1088547
-
Hacein-Bey-Abina, S., et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419. (Pubitemid 37296260)
-
(2003)
Science
, vol.302
, Issue.5644
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorensen, R.11
Forster, A.12
Fraser, P.13
Cohen, J.I.14
De Saint Basile, G.15
Alexander, I.16
Wintergerst, U.17
Frebourg, T.18
Aurias, A.19
Stoppa-Lyonnet, D.20
Romana, S.21
Radford-Weiss, I.22
Gross, F.23
Valensi, F.24
Delabesse, E.25
Macintyre, E.26
Sigaux, F.27
Soulier, J.28
Leiva, L.E.29
Wissler, M.30
Prinz, C.31
Rabbitts, T.H.32
Le Deist, F.33
Fischer, A.34
Cavazzana-Calvo, M.35
more..
-
19
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
-
Hacein-Bey-Abina, S., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
-
20
-
-
33847330241
-
Clinical experience using cultured epithelial autografts leads to an alternative methodology for transferring skin cells from the laboratory to the patient
-
Hernon, C.A., et al. (2006). Clinical experience using cultured epithelial autografts leads to an alternative methodology for transferring skin cells from the laboratory to the patient. Regen. Med. 1, 809-821.
-
(2006)
Regen. Med.
, vol.1
, pp. 809-821
-
-
Hernon, C.A.1
-
21
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
-
Howe, S.J., et al. (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 118, 3143-3150.
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 3143-3150
-
-
Howe, S.J.1
-
22
-
-
4444223718
-
Expression of LEKTI domains 6-9′ in the baculovirus expression system: Recombinant LEKTI domains 6-9′ inhibit trypsin and subtilisin A
-
DOI 10.1016/j.pep.2003.12.004
-
Jayakumar, A., et al. (2004). Expression of LEKTI domains 6-9¢ in the baculovirus expression system: recombinant LEKTI domains 6-9¢ inhibit trypsin and subtilisin A. Protein Expr. Purif. 35, 93-101. (Pubitemid 38497911)
-
(2004)
Protein Expression and Purification
, vol.35
, Issue.1
, pp. 93-101
-
-
Jayakumar, A.1
Kang, Y.2
Mitsudo, K.3
Henderson, Y.4
Frederick, M.J.5
Wang, M.6
El-Naggar, A.K.7
Marx, U.C.8
Briggs, K.9
Clayman, G.L.10
-
23
-
-
0036125197
-
Elevated stratum corneum hydrolytic activity in Netherton syndrome suggests an inhibitory regulation of desquamation by SPINK5-derived peptides
-
DOI 10.1046/j.0022-202x.2001.01663.x
-
Komatsu, N., et al. (2002). Elevated stratum corneum hydrolytic activity in Netherton syndrome suggests an inhibitory regulation of desquamation by SPINK5-derived peptides. J. Invest. Dermatol. 118, 436-443. (Pubitemid 34227160)
-
(2002)
Journal of Investigative Dermatology
, vol.118
, Issue.3
, pp. 436-443
-
-
Komatsu, N.1
Takata, M.2
Otsuki, N.3
Ohka, R.4
Amano, O.5
Takehara, K.6
Saijoh, K.7
-
24
-
-
0036145393
-
In vivo assessment of gene delivery to keratinocytes by lentiviral vectors
-
Kuhn, U., et al. (2002). In vivo assessment of gene delivery to keratinocytes by lentiviral vectors. J. Virol. 76, 1496-1504. (Pubitemid 34066449)
-
(2002)
Journal of Virology
, vol.76
, Issue.3
, pp. 1496-1504
-
-
Kuhn, U.1
Terunuma, A.2
Pfutzner, W.3
Foster, R.A.4
Vogel, J.C.5
-
25
-
-
33847256045
-
Progress and opportunities for tissue-engineered skin
-
MacNeil, S. (2007). Progress and opportunities for tissue-engineered skin. Nature 445, 874-880.
-
(2007)
Nature
, vol.445
, pp. 874-880
-
-
Macneil, S.1
-
26
-
-
0039702803
-
LEKTI, a novel 15-domain type of human serine proteinase inhibitor
-
Magert, HJ., et al. (1999). LEKTI, a novel 15-domain type of human serine proteinase inhibitor. J. Biol. Chem. 274, 21499-21502.
-
(1999)
J. Biol. Chem.
, vol.274
, pp. 21499-21502
-
-
Magert, H.J.1
-
27
-
-
33750337565
-
Functional lentiviral vectors for xeroderma pigmentosum gene therapy
-
DOI 10.1016/j.jbiotec.2006.05.016, PII S0168165606004706
-
Marchetto, M.C., Correa, R.G., Menck, C.F., and Muotri, A.R. (2006). Functional lentiviral vectors for xeroderma pigmento-sum gene therapy. J. Biotechnol. 126, 424-430. (Pubitemid 44634821)
-
(2006)
Journal of Biotechnology
, vol.126
, Issue.4
, pp. 424-430
-
-
Marchetto, M.C.N.1
Correa, R.G.2
Menck, C.F.M.3
Muotri, A.R.4
-
28
-
-
33845524625
-
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
-
DOI 10.1038/nm1504, PII NM1504
-
Mavilio, F., et al. (2006). Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat. Med. 12, 1397-1402. (Pubitemid 44924498)
-
(2006)
Nature Medicine
, vol.12
, Issue.12
, pp. 1397-1402
-
-
Mavilio, F.1
Pellegrini, G.2
Ferrari, S.3
Di Nunzio, F.4
Di Iorio, E.5
Recchia, A.6
Maruggi, G.7
Ferrari, G.8
Provasi, E.9
Bonini, C.10
Capurro, S.11
Conti, A.12
Magnoni, C.13
Giannetti, A.14
De Luca, M.15
-
29
-
-
0037426357
-
Inhibition of serine proteinases plasmin, trypsin, subtilisin A, cathepsin G, and elastase by LEKTI: A kinetic analysis
-
DOI 10.1021/bi027029v
-
Mitsudo, K., et al. (2003). Inhibition of serine proteinases plas-min, trypsin, subtilisin A, cathepsin G, and elastase by LEKTI: a kinetic analysis. Biochemistry 42, 3874-3881. (Pubitemid 36402680)
-
(2003)
Biochemistry
, vol.42
, Issue.13
, pp. 3874-3881
-
-
Mitsudo, K.1
Jayakumar, A.2
Henderson, Y.3
Frederick, M.J.4
Kang, Y.5
Wang, M.6
El-Naggar, A.K.7
Clayman, G.L.8
-
30
-
-
0036932834
-
In vivo correction of murine tyrosinemia type I by DNA-mediated transposition
-
DOI 10.1006/mthe.2002.0812
-
Montini, E., et al. (2002). In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Mol. Ther. 6, 759-769. (Pubitemid 36054309)
-
(2002)
Molecular Therapy
, vol.6
, Issue.6
, pp. 759-769
-
-
Montini, E.1
Held, P.K.2
Noll, M.3
Morcinek, N.4
Al-Dhalimy, M.5
Finegold, M.6
Yant, S.R.7
Kay, M.A.8
Grompe, M.9
-
31
-
-
33745108790
-
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
-
DOI 10.1038/nbt1216, PII N1216
-
Montini, E., et al. (2006). Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. 24, 687-696. (Pubitemid 43882120)
-
(2006)
Nature Biotechnology
, vol.24
, Issue.6
, pp. 687-696
-
-
Montini, E.1
Cesana, D.2
Schmidt, M.3
Sanvito, F.4
Ponzoni, M.5
Bartholomae, C.6
Sergi, L.S.7
Benedicenti, F.8
Ambrosi, A.9
Di Serio, C.10
Doglioni, C.11
Von Kalle, C.12
Naldini, L.13
-
32
-
-
3142656803
-
A new autologous keratinocyte dressing treatment for non-healing diabetic neuropathic foot ulcers
-
DOI 10.1111/j.1464-5491.2004.01166.x
-
Moustafa, M., et al. (2004). A new autologous keratinocyte dressing treatment for non-healing diabetic neuropathic foot ulcers. Diabet. Med. 21, 786-789. (Pubitemid 38916227)
-
(2004)
Diabetic Medicine
, vol.21
, Issue.7
, pp. 786-789
-
-
Moustafa, M.1
Simpson, C.2
Glover, M.3
Dawson, R.A.4
Tesfaye, S.5
Creagh, F.M.6
Haddow, D.7
Short, R.8
Heller, S.9
MacNeil, S.10
-
33
-
-
33645734405
-
Correction of X-linked chronic granulo-matous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott, M.G., et al. (2006). Correction of X-linked chronic granulo-matous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401-409.
-
(2006)
Nat. Med.
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
-
34
-
-
0001951363
-
Methods for clonal growth and serial cultuvation of normal human epidermal keratinocytes and mesothelial cells
-
R. Baserga, ed. (IRL Press, Oxford)
-
Rheinwald, J.G. (1989). Methods for clonal growth and serial cultuvation of normal human epidermal keratinocytes and mesothelial cells. In A Practical Approach. R. Baserga, ed. (IRL Press, Oxford), pp. 81-94.
-
(1989)
A Practical Approach
, pp. 81-94
-
-
Rheinwald, J.G.1
-
35
-
-
77955086483
-
Matriptase initiates activation of epidermal pro-kallikrein and disease onset in a mouse model of Netherton syndrome
-
Sales, K.U., et al. (2010). Matriptase initiates activation of epidermal pro-kallikrein and disease onset in a mouse model of Netherton syndrome. Nat. Genet. 42, 676-683.
-
(2010)
Nat. Genet.
, vol.42
, pp. 676-683
-
-
Sales, K.U.1
-
36
-
-
84856915092
-
Novel molecular therapies for heritable skin disorders
-
Uitto, J., Christiano, A.M., McLean, W.H., and McGrath, J.A. (2012). Novel molecular therapies for heritable skin disorders. J. Invest. Dermatol. 132, 820-828.
-
(2012)
J. Invest. Dermatol.
, vol.132
, pp. 820-828
-
-
Uitto, J.1
Christiano, A.M.2
McLean, W.H.3
McGrath, J.A.4
-
37
-
-
0036127719
-
The stem cell compartment in human interfollicular epidermis
-
DOI 10.1016/S0923-1811(02)00003-8, PII S0923181102000038
-
Watt, F.M. (2002). The stem cell compartment in human inter-follicular epidermis. J. Dermatol. Sci. 28, 173-180. (Pubitemid 34242651)
-
(2002)
Journal of Dermatological Science
, vol.28
, Issue.3
, pp. 173-180
-
-
Watt, F.M.1
|