Gene-based therapies for dominantly inherited retinopathies

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