Featured
-
-
Original Article |
Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease
- J L Roybal
- , M Endo
- & A W Flake
-
Original Article |
Non-viral gene therapy for GDNF production in RCS rat: the crucial role of the plasmid dose
- E Touchard
- , P Heiduschka
- & F Behar-Cohen
-
Original Article |
Optimal conditions for lentiviral transduction of engrafting human CD34+ cells
- N Uchida
- , M M Hsieh
- & J F Tisdale
-
Original Article |
Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE
- J Torres-Torronteras
- , A Gómez
- & R Martí
-
Original Article |
Improvement of cardiac fibrosis in dystrophic mice by rAAV9-mediated microdystrophin transduction
- J-H Shin
- , Y Nitahara-Kasahara
- & S Takeda
-
Original Article
| Open Access
AAV-mediated photoreceptor transduction of the pig cone-enriched retina
- C Mussolino
- , M della Corte
- & E M Surace
-
Original Article |
A tissue-specific, activation-inducible, lentiviral vector regulated by human CD40L proximal promoter sequences
- Z Romero
- , S Torres
- & I J Molina
-
Review |
Physiological and tissue-specific vectors for treatment of inherited diseases
- M G Toscano
- , Z Romero
- & F Martin
-
Original Article |
Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunity
- S Eghtesad
- , H Zheng
- & P R Clemens
-
Original Article |
CFTR expression and activity from the human CFTR locus in BAC vectors, with regulatory regions, isolated by a single-step procedure
- C Auriche
- , E G Di Domenico
- & F Ascenzioni
-
Short Communication |
Immune reactivity to type VII collagen: implications for gene therapy of recessive dystrophic epidermolysis bullosa
- V Pendaries
- , G Gasc
- & A Hovnanian
AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice