Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect.

Subjects

Subjects :

*GLYCOSAMINOGLYCANS
*GENETIC transformation
*BIOACCUMULATION
*CELL death
*COGNITIVE ability

Details

Language :

English

ISSN :

10967192

Volume :

120

Issue :

1/2

Database :

Academic Search Index

Journal :

Molecular Genetics & Metabolism

Publication Type :

Academic Journal

Accession number :

121104520

Full Text :

https://doi.org/10.1016/j.ymgme.2016.11.098