标题
Gene-based therapies for dominantly inherited retinopathies
作者
关键词
-
出版物
GENE THERAPY
Volume 19, Issue 2, Pages 137-144
出版商
Springer Nature
发表日期
2011-11-17
DOI
10.1038/gt.2011.172
参考文献
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。- Adeno-associated virus-mediated anti-DR5 chimeric antibody expression suppresses human tumor growth in nude mice
- (2011) Fujia Lv et al. CANCER LETTERS
- Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity
- (2011) Eva-Maria Handel et al. CURRENT GENE THERAPY
- Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
- (2011) Claudio Mussolino et al. EMBO Molecular Medicine
- Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa
- (2011) Sophia Millington-Ward et al. MOLECULAR THERAPY
- Zinc Finger Nucleases as tools to understand and treat human diseases
- (2010) David Davis et al. BMC Medicine
- Heat Shock Proteins as Suppressors of Accumulation of Toxic Prefibrillar Intermediates and Misfolded Proteins in Neurodegenerative Diseases
- (2010) Shigeki Arawaka et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Induction of Molecular Chaperones as a Therapeutic Strategy for the Polyglutamine Diseases
- (2010) Yoshitaka Nagai et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Heat Shock Proteins: Therapeutic Drug Targets for Chronic Neurodegeneration?
- (2010) M. Sajjad et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- The Expanding Universe of Neurotrophic Factors: Therapeutic Potential in Aging and Age-Associated Disorders
- (2010) C. Lanni et al. CURRENT PHARMACEUTICAL DESIGN
- AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa
- (2010) Haoyu Mao et al. HUMAN GENE THERAPY
- Efficient Transgene Reconstitution with Hybrid Dual AAV Vectors Carrying the Minimized Bridging Sequences
- (2010) Arkasubhra Ghosh et al. HUMAN GENE THERAPY
- Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90)
- (2010) Lawrence C.S. Tam et al. HUMAN MOLECULAR GENETICS
- Neuroprotective effect of overexpression of thioredoxin on photoreceptor degeneration in Tubby mice
- (2010) Li Kong et al. NEUROBIOLOGY OF DISEASE
- CNTF Induces Regeneration of Cone Outer Segments in a Rat Model of Retinal Degeneration
- (2010) Yiwen Li et al. PLoS One
- Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78
- (2010) M. S. Gorbatyuk et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Retinitis pigmentosa and allied conditions today: a paradigm of translational research
- (2010) Carmen Ayuso et al. Genome Medicine
- AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins
- (2009) A Georgiadis et al. GENE THERAPY
- Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene
- (2009) Arpad Palfi et al. HUMAN GENE THERAPY
- NADPH oxidase plays a central role in cone cell death in retinitis pigmentosa
- (2009) Shinichi Usui et al. JOURNAL OF NEUROCHEMISTRY
- Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
- (2009) Albert M Maguire et al. LANCET
- The Thioredoxin-like Protein Rod-derived Cone Viability Factor (RdCVFL) Interacts with TAU and Inhibits Its Phosphorylation in the Retina
- (2009) Ram Fridlich et al. MOLECULAR & CELLULAR PROTEOMICS
- Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy
- (2009) Naomi Chadderton et al. MOLECULAR THERAPY
- Functional Cone Rescue by RdCVF Protein in a Dominant Model of Retinitis Pigmentosa
- (2009) Ying Yang et al. MOLECULAR THERAPY
- Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
- (2009) Francesca Simonelli et al. MOLECULAR THERAPY
- Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
- (2009) Jiaxin Hu et al. NATURE BIOTECHNOLOGY
- Depleting Rac1 in mouse rod photoreceptors protects them from photo-oxidative stress without affecting their structure or function
- (2009) M. Haruta et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Genetic diagnosis by whole exome capture and massively parallel DNA sequencing
- (2009) Murim Choi et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Increased Expression of Glutathione Peroxidase 4 Strongly Protects Retina from Oxidative Damage
- (2008) Lili Lu et al. ANTIOXIDANTS & REDOX SIGNALING
- Tailoring the AAV vector capsid for gene therapy
- (2008) L H Vandenberghe et al. GENE THERAPY
- Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10)
- (2008) Lawrence C.S. Tam et al. HUMAN MOLECULAR GENETICS
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
- (2008) Kevin D Foust et al. NATURE BIOTECHNOLOGY
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
- (2008) A. V. Cideciyan et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Versatility of AAV vectors for retinal gene transfer
- (2007) Enrico M. Surace et al. VISION RESEARCH
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started