Gene-targeting pharmaceuticals for single-gene disorders.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R18, doi. 10.1093/hmg/ddv476
- By:
- Publication type:
- Article
Works matching IS 09646906 AND DT 2016 AND VI 25 AND IP R1
Results: 9
1
2
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R53, doi. 10.1093/hmg/ddv442
- By:
- Publication type:
- Article
3
Adeno-associated viral vectors for the treatment of hemophilia.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R36, doi. 10.1093/hmg/ddv475
- By:
- Publication type:
- Article
4
Gene therapy grows up (and moves out of the house).
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R1, doi. 10.1093/hmg/ddw060
- By:
- Publication type:
- Article
5
Gene therapy for lysosomal storage disorders: a good start.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R65, doi. 10.1093/hmg/ddv457
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- Publication type:
- Article
6
Progress and challenges in viral vector manufacturing.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R42, doi. 10.1093/hmg/ddv451
- By:
- Publication type:
- Article
7
Advances in treatment of achondroplasia and osteoarthritis.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R2, doi. 10.1093/hmg/ddv419
- By:
- Publication type:
- Article
8
Progress and prospects of gene therapy clinical trials for the muscular dystrophies.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R9, doi. 10.1093/hmg/ddv420
- By:
- Publication type:
- Article
9
Progress toward improved therapies for inborn errors of metabolism.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. R1, p. R27, doi. 10.1093/hmg/ddv418
- By:
- Publication type:
- Article