The General Hospital Corporation

Harvard College

General Hospital Corporation, the

Casey Maguire

Xandra O Breakefield

Johan Karl Olov Skog

David P Corey

Johan Skog

Leonora Balaj

Florian Eichler

Maryna V Ivanchenko

Killian S Hanlon

Bence Gyorgy

David Yellen

Shilpa Prabhakar

Bakhos A Tannous

Use of Microvesicles in the Treatment of Medical Conditions

Aspects of the disclosure relate to compositions and methods for treating hereditary hearing loss and/or vision loss, for example, due to Usher syndrome, Type 3A. In some embodiments, the disclosure provides a recombinant adeno-associated virus comprising: (i) an AAV-S capsid protein, and (ii) an isolated nucleic acid comprising a transgene (e.g., a transgene for expressing a clarin-1 protein). The present disclosure also provides methods of treating hereditary hearing loss and/or vision loss (e.g., Usher Syndrome, Type 3A) using the same.

Recombinant adeno associated virus encoding clarin-1 and uses thereof

Aspects of the disclosure relate to compositions and methods useful for treating hereditary hearing loss, for example, Usher syndrome type 3A or nonsyndromic hearing loss and deafness (DFNB1). In some embodiments, the disclosure provides isolated nucleic acids, vectors, and rAAV.9.PHP.B comprising a transgene encoding a Clarin-1 or a GJB2, and methods of treating hearing loss using the same.

AAV vectors encoding clarin-1 or GJB2 and uses thereof

The invention provides compositions and methods for treating tuberous sclerosis complex (TSC). In particular, provided are condensed tuberins (cTuberins), cTuberin nucleic acids, and recombinant adeno-associated viruses (rAAVs) carrying a cTuberin nucleic acid for treating a patient with TSC.

Gene therapy for tuberous sclerosis

This invention relates generally to populations of microvesicles containing or otherwise associated with viral particles, methods of producing these purified populations, and methods of using these purified populations in a variety of diagnostic, therapeutic and/or prophylactic indications.

Use of microvesicles in the treatment of medical conditions

Methods of the invention encompass delivery of nucleic acid sequences encoding ABCD1 for the treatment of X-linked Adrenoleukodystrophy (X-ALD), e.g., for Adrenomyeloneuropathy (AMN).

Intrathecal delivery of nucleic acid sequences encoding ABCD1 for treatment of adrenomyeloneuropathy

The specification provides methods for isolating extracellular vesicles. Extracellular vesicles can be efficiently isolated, e.g., from biological fluids or cell culture media, using a heparin-coated solid support.

Methods of isolating extracellular vesicles

Described herein is a variant of wild type Gaussia luciferase that catalyzes glow-type emission kinetics suited for high-throughput functional screening applications. Polypeptides, functional fragments, variants, and nucleic acids that encode the enhanced luciferase are further described. One such polypeptide corresponds to wild type Gaussia luciferase with a substitution mutation of I for M at position 43 of the mature peptide. Methods of use, assay systems and kits that contain the polypeptides and/or nucleic acids are further described.

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Arlington, MA, United States of America

Casey Maguire