GENE THERAPY FOR EYE PATHOLOGIES

• Main Authors: DANOS Olivier, VAN EVEREN Sherri, CURTISS Darin Thomas, PATEL Samir Maganbhai, GHANEKAR Avanti Arvind, YOO Jesse I, O''BERRY Anthony Ray, IRWIN-PACK Kim Rees
• Format: Patent
• Language: English; Spanish
• Published: 26.10.2021
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors. Composiciones y métodos para la administración de productos terapéuticos (tales como proteínas terapéuticas (anticuerpos), ARN terapéuticos (shARN, siARN y miARN) y aptámeros terapéuticos) a la retina/humor vítreo en los ojos de sujetos humanos para tratar patologías oculares, que implican vectores virales recombinantes tales como vectores de virus adenoasociado recombinante (rAAV).