Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB.

Subjects

Subjects :

*GENETIC transformation
*CLINICAL trials
*SANFILIPPO syndrome
*MUCOPOLYSACCHARIDOSIS
*CHILDREN'S health
*DIAGNOSIS
*THERAPEUTICS

Details

Language :

English

ISSN :

10967192

Volume :

117

Issue :

2

Database :

Academic Search Index

Journal :

Molecular Genetics & Metabolism

Publication Type :

Academic Journal

Accession number :

112948117

Full Text :

https://doi.org/10.1016/j.ymgme.2015.12.355