相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease
Andrew Feigin et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: A Randomized placebo-controlled phase 2B trial
Richard B. Moss et al.
HUMAN GENE THERAPY (2007)
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
Michael G. Kaplitt et al.
LANCET (2007)
Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC
Krystof S. Bankiewicz et al.
MOLECULAR THERAPY (2006)
A dose-ranging study of AAV-hAADC therapy in parkinsonian monkeys
John R. Forsayeth et al.
MOLECULAR THERAPY (2006)
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in leber congenital amaurosis
Samuel G. Jacobson et al.
HUMAN GENE THERAPY (2006)
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G. Jacobson et al.
MOLECULAR THERAPY (2006)
Immune responses to AAV in a phase I study for Canavan disease
S. W. J. McPhee et al.
JOURNAL OF GENE MEDICINE (2006)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy
TR Flotte et al.
HUMAN GENE THERAPY (2005)
Proteasome inhibition enhances AAV-mediated transgene expression in human synoviocytes in vitro and in vivo
K Jennings et al.
MOLECULAR THERAPY (2005)
Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis - A multicenter, double-blind, placebo-controlled trial
RB Moss et al.
CHEST (2004)
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study
TR Flotte et al.
HUMAN GENE THERAPY (2003)
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
CS Manno et al.
BLOOD (2003)
Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog
K Narfström et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2003)
In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement
K Narfstrom et al.
JOURNAL OF HEREDITY (2003)
Intramuscular administration of recombinant adeno-associated virus 2 α-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model:: Safety and immunologic aspects
SH Song et al.
MOLECULAR THERAPY (2002)
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
JA Wagner et al.
HUMAN GENE THERAPY (2002)
A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease
ML Aitken et al.
HUMAN GENE THERAPY (2001)
Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors
S Song et al.
GENE THERAPY (2001)
Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors
HJ Chao et al.
MOLECULAR THERAPY (2001)
Gene therapy restores vision in a canine model of childhood blindness
GM Acland et al.
NATURE GENETICS (2001)
Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors
HJ Chao et al.
MOLECULAR THERAPY (2000)
Practice parameter: Thymectomy for autoimmune myasthenia gravis (an evidence-based review) - Report of the Quality Standards Subcommittee of the American Academy of Neurology
GS Gronseth et al.
NEUROLOGY (2000)
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
MA Kay et al.
NATURE GENETICS (2000)
分享论文
