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US 2022/0054657 A1Published application
Gene therapy for retinitis pigmentosa
AssigneeGenzyme Corporation
InventorsCatherine O'RIORDAN, Matthew ADAMOWICZ
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Abstract
Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
Claims19
1-123. (canceled)
124:
- A nucleic acid comprising an intron derived from an MVM.
125:
- The nucleic acid of claim 124 wherein the MVM intron comprises SEQ ID NO:23.
126:
- The nucleic acid of claim 124, further comprising a promoter.
127:
- The nucleic acid of claim 126, wherein the promoter is located 5′ to the MVM intron.
128:
- An expression construct comprising the nucleic acid of claim 124.
129:
- A vector comprising the nucleic acid of claim 124.
130:
- A cell comprising the nucleic acid of claim 124.
131:
- The nucleic acid of claim 126, further comprising an enhancer.
132:
- The nucleic acid of claim 127, further comprising an enhancer.
133:
- An expression construct comprising the nucleic acid of claim 127.
134:
- An expression construct comprising the nucleic acid of claim 132.
135:
- A vector comprising the expression construct of claim 128.
136:
- A vector comprising the expression construct of claim 133.
137:
- A vector comprising the expression construct of claim 134.
138:
- A cell comprising the expression construct of claim 128.
139:
- A cell comprising the expression construct of claim 134.
140:
- A cell comprising the vector of claim 129.
141:
- A cell comprising the vector of claim 137.