Systemic AAV8-MTM1 gene therapy for X-linked myotubular myopathy

被引：0
|
作者：
Mack, D. L.
Poulard, K.
Goddard, M.
Snyder, J. M.
Grange, R. W.
Doering, J.
Strande, J. L.
Latournerie, V.
Veron, P.
Amor, F.
Denard, J.
Meng, H.
Yang, L.
Liu, F.
Buscara, L.
Le Bec, C.
Martin, S.
O'Callaghan, M.
Mingozzi, F.
Beggs, A. H.
Lawlor, M. W.
Mavilio, F.
Mavilio, F.
Childers, M. K.
Bello, A. Buj
机构：
[1] GENETHON, Paris, France
[2] Univ Washington, Seattle, WA 98195 USA
[3] Wake Forest Sch Med, Winston Salem, NC USA
[4] Virginia Polytech & State Univ, Blacksburg, VA USA
[5] Med Coll Wisconsin, Madison, WI USA
[6] Univ Florida, Gainesville, FL 32611 USA
[7] Audentes Therapeut, Kuopio, Finland
[8] Univ Paris 06, Genethon, F-75252 Paris 05, France
[9] Harvard Univ, Sch Med, Cambridge, MA 02138 USA
来源：
HUMAN GENE THERAPY | 2015年 / 26卷 / 10期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
P158
引用
收藏
页码：A73 / A74
页数：2
相关论文
共 50 条
  • [1] Gait Function in a Canine Model of X-Linked Myotubular Myopathy After Systemic Delivery of AAV8-MTM1
    Goddard, Melissa A.
    Mack, David L.
    Snyder, Jessica M.
    Lawlor, Michael W.
    Beggs, Alan H.
    Buj-Bello, Anna
    Childers, Martin K.
    MOLECULAR THERAPY, 2014, 22 : S153 - S153
  • [2] Ten-Year Durability of AAV8-MTM1 Gene Transfer in a Canine Model of X-Linked Myotubular Myopathy
    Luo, SiWei
    Snyder, Jessica M.
    Crudele, Julie
    Grange, Robert W.
    Mangin, Louise
    Renaud-Gabardos, Edith
    Lawlor, Michael
    Beggs, Alan H.
    Childers, Martin K.
    Bello, Ana Buj
    Mack, David L.
    MOLECULAR THERAPY, 2024, 32 (04) : 214 - 215
  • [3] Improved Respiratory Function Is Associated With Increased Survival After Systemic Delivery of AAV8-MTM1 in a Canine Model of X-Linked Myotubular Myopathy
    Goddard, Melissa A.
    Mack, David L.
    Snyder, Jessica M.
    Grange, Robert W.
    Lawlor, Michael W.
    Beggs, Alan H.
    Buj-Bello, Anna
    Childers, Martin K.
    MOLECULAR THERAPY, 2014, 22 : S153 - S154
  • [4] Respiratory Function in a Canine Model of X-Linked Myotubular Myopathy Following Regional Limb Infusion with Recombinant AAV8-MTM1
    Goddard, Melissa
    Joubert, Romain
    Poulard, Karine
    Grange, Robert
    Lawlor, Michael
    Moal, Christelle
    Jamet, Thibaud
    Daniele, Nathalie
    Martin, Samia
    Riviere, Christel
    Hammer, Caroline
    Van Wittenberghe, Laetitia
    Mitchell, Erin
    Vignaud, Alban
    Masurier, Carole
    Moullier, Philippe
    Beggs, Alan
    Buj-Bello, Anna
    Smith, Barbara
    Byrne, Barry
    Childers, Martin
    MOLECULAR THERAPY, 2013, 21 : S37 - S37
  • [5] Intravenous AAV8-MTM1 Prolongs Life and Ameliorates Severe Muscle Pathology in Mouse and Dog Models of X-Linked Myotubular Myopathy
    Childers, Martin K.
    Joubert, Romain
    Holder, Melanie N.
    Grange, Robert W.
    Doering, Jon
    Lawlor, Michael W.
    Moal, Christelle
    Jamet, Thibaud
    Daniele, Nathalie
    Martin, Samia
    Riviere, Christel
    Poppante, Kimberly
    Soker, Thomas
    Hammer, Caroline
    Van Wittenberghe, Laetitia
    Goddard, Melissa
    Mitchell, Erin
    Barber, Jane
    Furth, Mark E.
    Vignaud, Alban
    Masurier, Carole
    Moullier, Philippe
    Beggs, Alan H.
    Beggs, Alan H.
    Buj-Bello, Anna
    MOLECULAR THERAPY, 2013, 21 : S14 - S14
  • [6] Minimally Effective Dose of Systemic AAV8-MTM1 Needed To Prolong Survival and Correct Severe Muscle Pathology in a Canine Model of X-Linked Myotubular Myopathy
    Mack, David L.
    Poulard, Karine
    Goddard, Melissa
    Snyder, Jessica M.
    Grange, Robert W.
    Doering, Jon
    Czerniecki, Stefan
    Strande, Jennifer L.
    Latournerie, Virginie
    Veron, Philippe
    Meng, Hui
    Yang, Lin
    Liu, Fujun
    Le Bec, Christine
    Buscara, Laurine
    Martin, Samia
    Moullier, Philippe
    O'Callaghan, Michael
    Mingozzi, Federico
    Beggs, Alan H.
    Lawlor, Michael W.
    Buj-Bello, Anna
    Mavilio, Fulvio
    Childers, Martin K.
    MOLECULAR THERAPY, 2015, 23 : S201 - S201
  • [7] Intravenous infusion Of AAV8-MTM1 prolongs life and ameliorates severe muscle pathology in mouse and dog models of X-linked myotubular myopathy
    Childers, M. K.
    Joubert, R.
    Poulard, K.
    Holder, M. N.
    Grange, R. W.
    Doering, J.
    Lawlor, M. W.
    Moal, C.
    Jamet, T.
    Daniele, N.
    Martin, C.
    Riviere, C.
    Poppante, K.
    Soker, T.
    Hammer, C.
    Van Wittenberghe, L.
    Guan, X.
    Goddard, M.
    Mitchell, E.
    Barber, J.
    Furth, M. E.
    Vignaud, A.
    Masurier, C.
    Moullier, P.
    Beggs, A. H.
    Buj-Bello, A.
    NEUROMUSCULAR DISORDERS, 2013, 23 (9-10) : 760 - 760
  • [8] AAV8-MTM1 results in long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy
    Lawlor, Michael
    Mack, David
    Poulard, Karine
    Goddard, Melissa
    Snyder, Jessica
    Grange, Robert
    Doering, Jon
    Strande, Jennifer
    Latournerie, Virginie
    Veron, Philippe
    Meng, Hui
    Yang, Lin
    Liu, Fujun
    Buscara, Laurine
    Le Bec, Christine
    Martin, Samia
    O'Callaghan, Michael
    Mingozzi, Federico
    Beggs, Alan
    Mavilio, Fulvio
    Buj-Bello, Anna
    Childers, Martin
    JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, 2015, 74 (06): : 617 - 617
  • [9] Canine X-linked myotubular myopathy: A dose-finding study of systemic AAV8-MTM1 effects on muscle strength, gait, respiration, neuromuscular function and survival
    Mack, D. L.
    Goddard, M.
    Snyder, J. M.
    Doering, J.
    Lawlor, M. W.
    Moullier, P.
    O'Callaghan, M.
    Beggs, A. H.
    Mavilio, F.
    Poulard, K.
    Latoumerie, V.
    Buj-Bello, A.
    Childers, M.
    NEUROMUSCULAR DISORDERS, 2014, 24 (9-10) : 807 - 807
  • [10] Mutations in the MTM1 gene implicated in X-linked myotubular myopathy
    Laporte, J
    GuiraudChaumeil, C
    Vincent, MC
    Mandel, JL
    Tanner, SM
    LiechtiGallati, S
    WallgrenPettersson, C
    Dahl, N
    Kress, W
    Bolhuis, PA
    Fardeau, M
    Samson, F
    Bertini, E
    HUMAN MOLECULAR GENETICS, 1997, 6 (09) : 1505 - 1511
12345