Methods of delivering transgenes to the eye
AssigneeKrystal Biotech, Inc.
InventorSuma Krishnan
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Abstract
Provided herein are methods of delivering transgenes to an eye of a subject comprising the use of pharmaceutical compositions having one or more polynucleotides suitable for enhancing, increasing, augmenting, and/or supplementing the levels of the transgene in the eye. Also provided herein are methods of correcting vision loss in a subject in need thereof comprising the use of the pharmaceutical compositions described herein.
Claims21
A method of delivering a human transgene to an eye of a subject, the method comprising administering to the eye of the subject a pharmaceutical composition comprising:
- a) a replication-defective herpes simplex virus type-1 (HSV-1) comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises one or more polynucleotides comprising the human transgene;
- and
- b) a pharmaceutically acceptable carrier,
- wherein the pharmaceutical composition is administered topically or via injection to the eye of the subject,
- wherein the one or more polynucleotides comprising the human transgene are operably linked to a promoter suitable for transcription in a mammalian cell,
- wherein the subject has a disorder or disease of the eye associated with vision loss,
- wherein the recombinant HSV-1 genome comprises an inactivating mutation in one or both copies of the ICP4 HSV-1 gene, and
- wherein the recombinant HSV-1 genome comprises an inactivating mutation in the ICP22 HSV-1 gene.
A method of delivering a human transgene to an eye of a subject, the method comprising:
- providing a pharmaceutical composition comprising:
- a) a replication-defective HSV-1 comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises one or more polynucleotides comprising the human transgene, wherein the one or more polynucleotides comprising the human transgene are operably linked to a promoter suitable for transcription in a mammalian cell,
- wherein the recombinant HSV-1 genome comprises an inactivating mutation in one or both copies of the ICP4 HSV-1 gene,
- wherein the replication-defective HSV-1 is suitable for delivering the one or more polynucleotides comprising the human transgene to the epidermis and/or dermis of the subject;
- and
- b) a pharmaceutically acceptable carrier;
- and
- administering the pharmaceutical composition topically or via injection to the eye of the subject that has a disorder or disease of the eye associated with vision loss.
Dependencies are inferred from the claim text.