Optimized gene therapy targeting retinal cells
AssigneesResearch Institute At Nationwide Children's Hospital, Sanford Research Company
InventorsKathrin Christine Meyer, Shibi Likhite, Brian K. Kaspar, Jill M. Weimer
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Abstract
The present disclosure relates to methods of targeting specific cell types within the retina using optimized gene therapy vectors. In particular, the disclosure provides gene therapy vectors to specifically target retinal cells and methods of treating visual impairment, retinal degeneration and vision-related disorders such as CLN disease.
Claims51
A method of delivering a transgene to a retinal cell in a subject comprising administering a gene therapy vector encoding the transgene, wherein the gene therapy vector is administered to the subject using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
A method of treating visual impairment or a vision-related disorder in a subject comprising administering a gene therapy vector encoding a transgene to the subject, wherein the gene therapy vector is administered using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
A method of treating Batten disease in a subject comprising administering to the subject a gene therapy vector comprising a polynucleotide encoding a CLN polypeptide, wherein the gene therapy vector is administered using sub-retinal delivery, intravitreous delivery or intrathecal delivery.
A composition for delivering a transgene to a retinal cell in a subject, wherein the composition comprises a gene therapy vector encoding the transgene, wherein the composition is formulated for administering the gene therapy vector using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
A composition for treating visual impairment or a vision-related disorder in a subject, wherein the composition comprises a gene therapy vector encoding a transgene to the subject, wherein the composition is formulated for administering the gene therapy vector using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
A composition for treating Batten disease in a subject, wherein the composition comprises a gene therapy vector comprising a polynucleotide encoding a CLN polypeptide, wherein the composition is formulated for administering the gene therapy vector using sub-retinal delivery, intravitreous delivery or intrathecal delivery.
Use of gene therapy for the preparation of a medicament for delivering a transgene to a retinal cell in a subject, wherein the medicament comprises a gene therapy vector encoding the transgene, and wherein the medicament is formulated for administering the gene therapy vector using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
Use of a gene therapy vector the preparation of a medicament for treating visual impairment or a vision-related disorder in a subject, wherein the medicament comprises a gene therapy vector encoding a transgene, wherein the medicament is formulated for administering the gene therapy vector using local intravenous delivery, sub-retinal delivery, intravitreous delivery or intrathecal delivery.
Use of a gene therapy vector for the preparation of a medicament for treating Batten disease in a subject, wherein the medicament comprises a gene therapy vector comprising a polynucleotide encoding a CLN polypeptide, and wherein the medicament is formulated for administering the gene therapy vector using sub-retinal delivery, intravitreous delivery or intrathecal delivery.
Dependencies are inferred from the claim text.