The General Hospital Corporation

Children's Medical Center Corporation

Other

Northeastern Ohio University

Yale University

Dana-Farber-Cancer Institute Inc.

Boston University

Stichting Katholieke Universiteit

The General Hosptial Corporation

The General Hospital Corpoation

The Eunice Kennedy Shriver Center for Mental Retardation

Xandra O Breakefield

Johan Karl Olov Skog

Robert L Martuza

Evan Y Snyder

Mikkel Noerholm

E Antonio Chiocca

Karen S Aboody

Laurie J Ozelius

Leonora Balaj

James F Gusella

Susan A Slaugenhaupt

Casey Maguire

Anat Blumenfeld

Bob Carter

Yoshinaga Saeki

Marion Priscilla Short

Ling Chen

Mark Suter

Leileata M Russo

Gosse Jan Adema

Johan Skog

Mehmet Fatih Bolukbasi

Dennis Brown

William P Lynch

Ulrich Herrlinger

Michael B Rosenberg

Mathias Ackermann

Gary J Brenner

Thurman Wheeler

David J Waxman

Matthew A Spear

Cornel Fraefel

Giulia Fulci

Shilpa Prabhakar

Ming X Wei

Alfred I Geller

H G Brunner

Frances I Smith

David R Jacoby

Alice B Brown

David Yellen

Edward Hawrot

Okay Saydam

Arda Mizrak

Manish Aghi

Christof M Kramm

Miguel Sena-Esteves

Ken Shortman

Kurt Tobler

Yun-Pung Hsu

Kevin C Miranda

Johan Karl Skog

Kevin Miranda

Leileata Russo

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

Described herein are methods for diagnosing and monitoring subjects with diseases associated with aberrant splicing, based upon detecting properly spliced isoforms and mis-spliced isoforms in a urine sample from the subject.

Extracellular mRNA markers of muscular dystrophies in human urine

The invention concerns gene signatures obtained from microvesicles and a method of applying these gene signatures in helping to determine a biological condition. The determination of a biological condition may aid, for example, the diagnosis, prognosis, and therapy treatment selection for disease in a subject.

Use of microvesicles in analyzing nucleic acid profiles

Microvesicles are small membrane vesicles that either shed or bud off eukarotic cells. Analysis of the nucleic acid content of microvesicles may be useful in detecting the presence or absence of genetic aberrations. This invention discloses novel methods of diagnosing, prognosing, monitoring, or treating a disease, such as cancer, or other medical condition in a subject involving analyzing one or more nucleic acids contained within an isolated microvesicle for the presence or absence of one or more Kras genetic aberrations.

Use of microvesicles in analyzing mutations

Disclosed herein are methods for assaying a biological sample from a subject by analyzing components of microvesicle fractions in aid of risk, diagnosis, prognosis or monitoring of, or directing treatment of the subject for, a disease or other medical condition in the subject. Also disclosed are methods of treatment and identifying biomarkers using a microvesicle fraction of a subject. Kits, pharmaceutical compositions, and profiles related to the methods are also disclosed.

Cancer-related biological materials in microvesicles

Disclosed herein is an isolated nucleic acid molecule comprising a first nucleic acid sequence 5'-ACCCTGCCGCCTGGACTCCGCCTGT-3&#x2032; (SEQ ID NO: 22), or a functional variant thereof, operably linked to a second, heterologous nucleic acid sequence. The isolated nucleic acid molecule can be DNA (in an expression vector) and RNA (mRNA, shRNA, orncRNA). Also disclosed is a microvesicle comprising the nucleic acid molecule and a microvesicle preparation comprising the microvesicle. Also disclosed is an in vitro method of producing a microvesicle preparation enriched for a specific RNA sequence by transfecting cells with the nucleic acid sequence, and isolating microvesicles generated therefrom. Methods of delivering therapeutic RNA to a subject are also disclosed.

Targeting RNAs to microvesicles

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 are compositions and methods for treating, inhibiting, preventing, reducing the severity of and/or reducing the progression of schwannoma, as well as any disease or physiological disease-state in which schwannoma plays a role. The compositions comprise an rAAV vector that includes a caspase-1 gene or a variant thereof and a Schwann cell specific promoter.

AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors

The present invention is based upon a surprising finding that stem cells, more particularly neural stem cells, can migrate throughout a brain tumor and track metastatic brain tumor cells. The invention provides a method for treating brain tumors by administering genetically engineered neural stem cells in an individual affected by brain tumors. The invention also provides a method of preparing genetically engineered neural stem cells and a composition comprising genetically engineered neural stem cells in a pharmaceutically acceptable carrier.

Neural stem cells and use thereof for brain tumor therapy

The present invention relates to the field of cellular and molecular therapy with modified (genetically or growth factor engineered) and unmodified stem cells (SCs). More particularly, the invention relates to a method of systemic treatment of central nervous system (CNS) and other tumors in both intracranial/intraspinal and extracranial/extraspinal sites, using neural stem cells (NSCs), a prototype for solid organ, non-hematopoietic stem cells.

Systemic gene delivery vehicles for the treatment of tumors

The present invention relates to methods of detecting mutations and polymorphisms in the torsin gene, torsin-related genes, methods of detecting neuronal diseases mediated by these mutations and polymorphisms and nucleic acids used in these methods.

Torsin, torsin-related genes and methods of detecting neuronal disease

Methods of killing neoplastic cells are provided. The invention relates to the use of folylpolyglutamyl synthetase (FPGS) gene transfer to enhance the sensitivity of several types of tumor cells to polyglutamylatable antifolate drugs, such as methotrexate (MTX) and edatrexate (EDX).

Folypolyglutamyl synthetase gene transfer to enhance antifolate

The present invention relates to a triple hybrid vector amplicon system comprising genetic elements derived from Herpes Simplex Virus (HSV), Epstein-Barr Virus (EBV) or Adeno-Associated Virus (AAV), and a retrovirus. The vector was developed to stably transform cells, both in culture or in vivo, into retrovirus packaging cells in a single step. This step can be accomplished both by transfection using liposomes, electroporation, calcium phosphate, or any other methodology used to transfer naked or complexed DNA into cells or by infection when the vector is packaged as an amplicon vector in HSV virions.

Triple hybrid amplicon vector systems to generate retroviral packaging lines

Growing community of inventors

Newton, MA, United States of America

Xandra O Breakefield