Adeno-associated Viral Vectors do not Efficiently Target Muscle Satellite Cells

Adeno-associated viral (AAV) vectors are becoming an important tool for gene therapy of numerous genetic and other disorders. Several recombinant AAV vectors (rAAV) have the ability to transduce striated muscles in a variety of animals following intramuscular and intravascular administration, and have attracted widespread interest for therapy of muscle disorders such as the muscular dystrophies. However, most studies have focused on the ability to transduce mature muscle cells, and have not examined the ability to target myogenic stem cells such as skeletal muscle satellite cells.

Here we examined the relative ability of rAAV vectors derived from AAV6 to target myoblasts, myocytes, and myotubes in culture and satellite cells and myofibers in vivo. AAV vectors are able to transduce proliferating myoblasts in culture, albeit with reduced efficiency relative to postmitotic myocytes and myotubes. In contrast, quiescent satellite cells are refractory to transduction in adult mice.

These results suggest that while muscle disorders characterized by myofiber regeneration can be slowed or halted by AAV transduction, little if any vector transduction can be obtained in myogenic stems cells that might other wise support ongoing muscle regeneration.

Topics & Tags

Read full article:

Arnett ALH, Konieczny P, Ramos JN, Hall J, Odom G, Yablonka-Reuveni Z, Chamberlain JR, Chamberlain JS:
Adeno-associated Viral Vectors do not Efficiently Target Muscle Satellite Cells

Mol Ther Methods Clin Dev. 1: 14038 (2014); doi: 10.1038/mtm.2014.38

Related Pages

Interested to know more?

Talk to our experts. We are happy to answer all your questions and concerns.

Contact Us

Do you prefer personal consulting?

  • Leica Microsystems Inc.
    1700 Leider Lane
    Buffalo Grove, IL 60089 United States
    Office Phone : +1 800 248 0123
    Service Phone : 1 800 248 0223
    Fax : +1 847-236-3009

You will find a more detailed list of local contacts here.