Scott Uretsky
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View article: 267. Phase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4
267. Phase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4 Open
Introduction Leber Hereditary Optic Neuropathy (LHON) is a rare mitochondrial genetic disorder predominantly affecting young males. Affected patients experience bilateral severe central vision loss. Currently no therapy is approved in the …
View article: 83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patients
83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patients Open
GenSight Biologics develops a rAAV2/2-ND4 vector (GS010) administered via intravitreal route (IVI) against vision loss in LHON patients bearing ND4 mutation. Gene therapy Adeno Associated Viral vectors are known to trigger host immune resp…