rAAV2/2-ND4 for the Treatment of Leber Hereditary Optic Neuropathy (LHON): 72-Week Data from the REVERSE Phase III Clinical Trial

被引：0

作者：

Yu-Wai-Man, Patrick ^{[1
,2
]}

Moster, Mark ^{[3
,4
,5
]}

Sadun, Alfredo A. ^{[6
,7
]}

Klopstock, Thomas ^{[8
]}

Vignal-Clermont, Catherine ^{[9
,10
]}

Newman, Nancy J. ^{[11
,12
,13
]}

Sergott, Robert C. ^{[14
]}

Carelli, Valerio ^{[15
,16
]}

Chevalier, Caroline ^{[17
]}

Blouin, Laure ^{[17
]}

Taiel, Magali ^{[17
]}

Katz, Barrett ^{[17
]}

Sahel, Jose Alain ^{[18
,19
]}

机构：

[1] Univ Cambridge, Dept Clin Neurosci, Cambridge, England

[2] Moorfields Eye Hosp, London, England

[3] Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USA

[4] Thomas Jefferson Univ, Dept Neurol, Sidney Kimmel Med Coll, Philadelphia, PA 19107 USA

[5] Thomas Jefferson Univ, Dept Ophthalmol, Sidney Kimmel Med Coll, Philadelphia, PA 19107 USA

[6] Doheny Eye Inst, 1355 San Pablo St, Los Angeles, CA 90033 USA

[7] Univ Calif Los Angeles, Dept Ophthalmol, Los Angeles, CA USA

[8] Ludwig Maximilians Univ Munchen, Friedrich Baur Inst, Dept Neurol, Munich, Germany

[9] Ctr Hosp Natl Ophtalmol Quinze Vingts, Paris, France

[10] Fdn Ophtalmol Rothschild, Paris, France

[11] Emory Univ, Sch Med, Dept Ophthalmol, Atlanta, GA 30322 USA

[12] Emory Univ, Sch Med, Dept Neurol, Atlanta, GA 30322 USA

[13] Emory Univ, Sch Med, Dept Neurol Surg, Atlanta, GA USA

[14] Wills Eye Hosp & Res Inst, Neuroophthalmol Serv, Philadelphia, PA USA

[15] Bellaria Hosp, IRCCS Inst Neurol Sci Bologna, Bologna, Italy

[16] Univ Bologna, Neurol Unit, Dept Biomed & Neuromotor Sci DIBINEM, Bologna, Italy

[17] GenSight Biol, Paris, France

[18] Sorbonne Univ, Inst Vis, Paris, France

[19] Univ Pittsburgh, Sch Med, Dept Ophthalmol, Pittsburgh, PA 15261 USA

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2019年 / 60卷 / 09期

关键词：

D O I：

暂无

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

4806

引用

页数：4

共 39 条

[1] rAAV2/2-ND4 for the Treatment of LHON:.72-week Data from the REVERSE Phase III Clinical Trial
Moster, Mark L.
Sadun, Alfredo
Klopstock, Thomas
Vignal-Clermont, Catherine
Newman, Nancy J.
Carelli, Valerio
Yu-Wai-Man, Patrick
Sahel, Jose-Alain
Katz, Barrett
NEUROLOGY, 2019, 92 (15)
[2] rAAV2/2-ND4 for the Treatment of Leber Hereditary Optic Neuropathy (LHON): Final Results from the RESCUE and REVERSE Phase III Clinical Trials and Experimental Data in Nonhuman Primates to Support a Bilateral Effect
Moster, Mark
Sadun, Alfredo
Klopstock, Thomas
Newman, Nancy
Vignal-Clermont, Catherine
Carelli, Valerio
Yu-Wai-Man, Patrick
Biousse, Valerie
Sergott, Robert
Katz, Barrett
DeBusk, Adam
Blouin, Laure
Chevalier, Caroline
Burguiere, Pierre
Taiel, Magali
Sahel, Jose-Alain
NEUROLOGY, 2020, 94 (15)
[3] Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy
Vignal, Catherine
Uretsky, Scott
Fitoussi, Serge
Galy, Anne
Blouin, Laure
Girmens, Jean-Francois
Bidot, Samuel
Thomasson, Nitza
Bouquet, Celine
Valero, Sonia
Meunier, Sandrine
Combal, Jean-Philippe
Gilly, Bernard
Katz, Barrett
Sahel, Jose-Alain
OPHTHALMOLOGY, 2018, 125 (06) : 945 - 947
[4] Allotopic Gene Therapy for ND4 Leber Hereditary Optic Neuropathy with rAAV2/2-ND4 (GS010): From Pre-Clinical Development to Phase III
Uretsky, Scott
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
[5] Evaluation of the efficacy of rAAV2/2-ND4 gene therapy for Leber hereditary optic neuropathy compared with the natural history
Yu-Wai-Man, Patrick
Newman, Nancy J.
Carelli, Valerio
Moster, Mark
Vignal-Clermont, Catherine
Sadun, Alfredo A.
La Morgia, Chiara
Bandello, Francesco
Campillo, Lorena Castillo
Leruez, Stephanie
Cestari, Dean
Foroozan, Robert
Karanjia, Rustum
Blouin, Laure
Taiel, Magali
Sahel, Jose Alain
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)
[6] Relationship between immune response and ocular inflammation after intravitreal injection of rAAV2/2-ND4 (GS010) in Leber hereditary optic neuropathy (LHON) patients
Clermont, C. Vignal
Bouquet, C.
Uretsky, S.
Galy, A.
Thomasson, N.
Fitoussi, S.
Combal, J. P.
Sahel, J.
ACTA OPHTHALMOLOGICA, 2016, 94
[7] Intravitreal injection of rAAV2/2-ND4 (GSO10): immune responses and ocular inflammation in non-human primates and patients with Leber Hereditary Optic Neuropathy (LHON)
Thomasson, Nitza
Bouquet, Celine
Galy, Anne
Fitoussi, Serge
Meunier, Sandrine
Uretsky, Scott
Vignal, Catherine
Sahel, Jose
Combal, Jean Philppe
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
[8] Evaluation of rAAV2/2-ND4 Gene Therapy Efficacy in Leber Hereditary Optic Neuropathy Using an External Control Group of Untreated Patients
Newman, Nancy
Yu-Wai-Man, Patrick
Carelli, Valerio
Moster, Mark
Vignal-Clermont, Catherine
Sadun, Alfredo
La Morgia, Chiara
Bandello, Francesco
Campillo, Lorena Castillo
Leruez, Stephanie
Cestari, Dean
Foroozan, Rod
Karanjia, Rustum
Blouin, Laure
Roux, Michel
Taiel, Magali
Sahel, Jose-Alain
NEUROLOGY, 2021, 96 (15)
[9] Intravitreal rAAV2/2-ND4 (GS010): A gene therapy for vision loss in Leber's Hereditary Optic Neuropathy (LHON) caused by the G11778A ND4 mitochondrial mutation
Uretsky, Scott
Vignal, Catherine
Thomasson, Nitza
Bouquet, Celine
Galy, Anne
Combal, Jean Philipppe
Fitoussi, Serge
Sahel, Jose-Alain
NEUROLOGY, 2017, 88
[10] Lenadogene nolparvovec rAAV2 gene therapy vector encoding ND4 Treatment of Leber hereditary optic neuropathy
Desideri, Lorenzo Ferro
Traverso, Carlo Enrico
Iester, Michele
DRUGS OF THE FUTURE, 2022, 47 (06) : 429 - 433

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