Extracellular Vesicles and Therapeutic Vehicles for Myotonic Dystrophy

Abstract

Scientific objective: Our goal is to test a novel method of gene therapy that uses vesicles or "bubble-like" structures to carry the therapeutic gene and protect it from damage by the recipient's immune system. Rationale: One form of gene therapy involves the use of a tool known as an adeno-associated viral (AAV) vector to deliver or carry the therapeutic gene to the muscle fibers. Sometimes a person's immune system can make gene therapy difficult by attacking the AAV vector, thereby reducing the delivery of the AAV vector. A method that enhances delivery of the AAV vector to muscle tissue and that is resistant to the immune system is needed. Topic Area: Myotonic dystrophy; Area of Encouragement: Development and/or testing of novel and/or innovative treatments, including those utilizing gene editing or silencing.

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Document Details

Document Type
Technical Report
Publication Date
Mar 01, 2021
Accession Number
AD1133896

Entities

People

  • Thurman Wheeler

Organizations

  • Massachusetts General Hospital

Tags

DTIC Thesaurus Topics

  • Acquisition
  • Biological Markers
  • Bioluminescence
  • Biomedical Research
  • Gel Electrophoresis
  • Gene Expression
  • Gene Therapy
  • Health Services
  • Hospitals
  • Immune System
  • Intramuscular Injections
  • Massachusetts
  • Muscles
  • Skeletal Muscle
  • Standards
  • Therapy
  • Tissues

Fields of Study

  • Medicine

Readers

  • Molecular Genetics
  • Oncology (Cancer Research).
  • Systems Analysis and Design

Technology Areas

  • Biotechnology