First-In-Human Intrathecal Gene Transfer Study for Giant Axonal Neuropathy: Interim Analysis of Efficacy and Review of Safety

被引：0

作者：

Bharucha-Goebel, Diana ^{[1
]}

Saade, Dimah ^{[1
]}

Jain, Mina ^{[2
]}

Waite, Melissa ^{[2
]}

Norato, Gina ^{[3
]}

Cheung, Ken ^{[1
]}

Foley, Reghan ^{[1
]}

Soldatos, Ariane ^{[4
]}

Rybin, Denis ^{[5
]}

Lehky, Tanya ^{[6
]}

Hu, Ying ^{[1
]}

Whitehead, Matthew ^{[7
]}

Del Hoyo, Roberto Calcedo ^{[8
]}

Jacobson, Steven ^{[9
]}

Nath, Avindra ^{[9
]}

Grieger, Joshua ^{[10
]}

Samulski, Jude ^{[11
]}

Gray, Steven ^{[12
]}

Bonnemann, Carsten ^{[1
]}

机构：

[1] NINDS, Neurogenet Branch, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[2] NINDS, Rehabil Dept, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[3] NINDS, Off Biostat, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[4] NINDS, Pediat Neurol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[5] Pfizer Inc, Cambridge, MA USA

[6] NINDS, Neurophysiol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[7] Childrens Natl Hlth Syst, Neuroradiol, Washington, DC USA

[8] Univ Penn, Philadelphia, PA 19104 USA

[9] NINDS, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[10] Asklepios Biopharmaceut Inc, Chapel Hill, NC USA

[11] Bamboo Therapeut, Chapel Hill, NC USA

[12] UT Southwestern, Pediat, Dallas, TX USA

来源：

MOLECULAR THERAPY | 2018年 / 26卷 / 05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：

引用

页码：32 / 33

页数：2

共 50 条

[1] Review of Safety and Interim Analysis of Efficacy of a First-in-Human Intrathecal Gene Transfer Study for Giant Axonal Neuropathy
Saade, Dimah
Bharucha-Goebel, Diana
Jain, Mina
Waite, Melissa
Norato, Gina
Cheung, Kuen
Foley, A. Reghan
Soldatos, Ariane
Rybin, Denis
Lehky, Tanya
Hu, Ying
Whitehead, Matthew
Del Hoyo, Roberto Calcedo
Jacobson, Steven
Nath, Avindra
Grieger, Joshua
Samulski, R. Jude
Gray, Steven J.
Bonnemann, Carsten G.
MOLECULAR THERAPY, 2019, 27 (04) : 190 - 190
[2] First-in-human intrathecal gene transfer study for giant axonal neuropathy: review of safety, immunologic responses and interim analysis of efficacy
Saade, D.
Bharucha-Goebel, D.
Jain, M.
Waite, M.
Norato, G.
Cheung, K.
Foley, A. R.
Soldatos, A.
Rybin, D.
Lehky, T.
Ying, H.
Whitehead, M.
Del Hoyo, R. Calcedo
Jacobson, S.
Leibovitch, E.
Nath, A.
Grieger, J.
Samulski, R.
Gray, S.
Bonnemann, C.
NEUROMUSCULAR DISORDERS, 2018, 28 : S91 - S92
[3] Review of Safety and Interim Analysis of Efficacy in the First in Human Intrathecal Gene Transfer Trial for Giant Axonal Neuropathy
Saade, Dimah
Bharucha-Goebel, Diana
Jain, Mina
Waite, Melissa
Norato, Gina
Cheung, Ken
Foley, A. Reghan
Paredes, Eduardo
Soldatos, Ariane
Rybin, Denis
Lehky, Tanya
Charnas, Lawrence
Hu, Ying
Whitehead, Matthew
Nath, Avindra
Zein, Wadih
Grieger, Josh
Samulski, R. Jude
Gray, Steven J.
Bonnemann, Carsten G.
MOLECULAR THERAPY, 2020, 28 (04) : 273 - 273
[4] First-in-human intrathecal gene transfer study for giant axonal neuropathy: Three-year interim evaluation of safety and efficacy
Bharucha-Goebel, D.
Saade, D.
Jain, M.
Norato, G.
Rybin, D.
Cheung, K.
Waite, M.
Paredes, E.
Foley, A.
Lehky, T.
Hu, Y.
Del Hoyo, R. Calcedo
Chichester, J.
Jacobson, S.
Nath, A.
Charnas, L.
Samulski, R.
Gray, S.
Bonnemann, C.
NEUROMUSCULAR DISORDERS, 2020, 30 : S78 - S78
[5] First-In-Human Intrathecal Gene Transfer Study for Giant Axonal Neuropathy: Three-Year Study Review of Safety, Immunologic Responses and Interim Analysis of Efficacy
Bharucha-Goebel, D.
Saade, D.
Jain, M.
Waite, M.
Norato, G.
Cheung, K.
Foley, A. R.
Soldatos, A.
Rybin, D.
Lehky, T.
Del Hoyo, Calcedo R.
Jacobson, S.
Samulski, R. J.
Gray, S.
Bonnemann, C.
ANNALS OF NEUROLOGY, 2019, 86 : S168 - S168
[6] First-in-human intrathecal gene transfer study for giant axonal neuropathy: Preliminary review of long-term efficacy and safety
Bharucha-Goebel, D.
Saade, D.
Todd, J.
Norato, G.
Jain, M.
Waite, M.
Armao, D.
Foley, A.
Lehky, T.
Averion, G.
Hu, Y.
Mohassel, P.
Hoke, A.
DeLong, T.
Acquaye, N.
Hinkley, L.
Chichester, J.
Mendoza, C.
Soldatos, A.
Gray, S.
Bonnemann, C.
NEUROMUSCULAR DISORDERS, 2022, 32 : S94 - S94
[7] Review of CSF and Peripheral Immune Responses Following Intrathecal Gene Transfer for Giant Axonal Neuropathy Intrathecal Gene Transfer for Giant Axonal Neuropathy
Bharucha-Goebel, Diana
Foley, A. Reghan
Soldatos, Ariane
Hu, Ying
Hewitt, Matthew
Del Hoyo, Roberto Calcedo
Jacobson, Steven
Nath, Avindra
Bailey, Rachel M.
Grieger, Josh
Samulski, R. Jude
Bollard, Catherine
Gray, Steven J.
Bonnemann, Carsten G.
MOLECULAR THERAPY, 2017, 25 (05) : 240 - 241
[8] Review of ophthalmologic findings following intrathecal gene transfer for giant axonal neuropathy
Saade, D.
Bharucha-Goebel, D.
Zein, W.
Norato, G.
Rybin, D.
Cheung, K.
Charnas, L.
Paredes, E.
Inati, S.
Foley, A.
Gray, S.
Bonnemann, C.
NEUROMUSCULAR DISORDERS, 2020, 30 : S78 - S78
[9] First in human IT gene transfer for GAN: Review of safety, immunology & interim analysis of efficacy
Saade, Dimah
Bharucha-Goebel, Diana
Jain, Mina
Waite, Melissa
Norato, Gina
Cheung, Ken
Foley, A. Reghan
Soldatos, Ariane
Rybin, Denis
Lehky, Tanya
Hu, Ying
Whitehead, Matthew
Del Hoyo, Roberto Calcedo
Jacobson, Steven
Nath, Avrinda
Grieger, Joshua
Samulski, R. Jude
Gray, Steven
Bonnemann, Carsten
JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2018, 23 (04) : 325 - 326
[10] Postmortem Assessment of Vector Biodistribution in the First-in-Human Intrathecal scAAV9 Gene Therapy Trial for Giant Axonal Neuropathy
Gray, Steven J.
Bailey, Rachel M.
Bharucha-Goebel, Diana
Foley, A. Reghan
Zaric, Violeta
Crawford, Thomas O.
Armao, Diane
Sumner, Charlotte J.
Hoke, Ahmet
Chen, Liam
Grieger, Joshua C.
Samulski, R. Jude
Mohassel, Payam
Hooper, Jody E.
Bonnemann, Carsten
MOLECULAR THERAPY, 2017, 25 (05) : 139 - 139

← 1 2 3 4 5 →