4.0 Review

Gene therapy in inherited retinal degenerative diseases, a review

Related references

Note: Only part of the references are listed.
Article Medicine, Research & Experimental

Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach

Simon M. Petersen-Jones et al.

JOURNAL OF CLINICAL INVESTIGATION (2018)

Review Ophthalmology

Caring for Hereditary Childhood Retinal Blindness

Ruben Jauregui et al.

ASIA-PACIFIC JOURNAL OF OPHTHALMOLOGY (2018)

Article Ophthalmology

CLINICAL PRESENTATION AND DISEASE COURSE OF USHER SYNDROME BECAUSE OF MUTATIONS IN MYO7A OR USH2A

Francesco Testa et al.

RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES (2017)

Article Neurosciences

Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

Freya M. Mowat et al.

FRONTIERS IN NEUROSCIENCE (2017)

Article Ophthalmology

Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates

Immanuel P. Seitz et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2017)

Article Ophthalmology

A Single Wide-Field OCT Protocol Can Provide Compelling Information for the Diagnosis of Early Glaucoma

Donald C. Hood et al.

TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2016)

Article Ophthalmology

Visual Function and Central Retinal Structure in Choroideremia

Elise Heon et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2016)

Review Ophthalmology

Retinitis Pigmentosa: Progress and Perspective

Qingjiong Zhang

ASIA-PACIFIC JOURNAL OF OPHTHALMOLOGY (2016)

Review Biochemistry & Molecular Biology

Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities

Pranav Mathur et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2015)

Article Biotechnology & Applied Microbiology

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Wen-Tao Deng et al.

HUMAN GENE THERAPY (2015)

Article Medicine, Research & Experimental

Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

Jingxing Ou et al.

JOURNAL OF CLINICAL INVESTIGATION (2015)

Article Biochemistry & Molecular Biology

Retinoschisin gene therapy in photoreceptors, Muller glia or all retinal cells in the Rs1h-/- mouse

L. C. Byrne et al.

GENE THERAPY (2014)

Editorial Material Biochemistry & Molecular Biology

Clinical utility gene card for: Achromatopsia - update 2013

Susanne Kohl et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2013)

Article Biochemistry & Molecular Biology

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

Katherine J. Wert et al.

HUMAN MOLECULAR GENETICS (2013)

Article Multidisciplinary Sciences

A Large Animal Model for CNGB1 Autosomal Recessive Retinitis Pigmentosa

Paige A. Winkler et al.

PLOS ONE (2013)

Article Genetics & Heredity

A Nonsense Mutation in PDE6H Causes Autosomal-Recessive Incomplete Achromatopsia

Susanne Kohl et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2012)

Article Biochemistry & Molecular Biology

Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa

Susanne Koch et al.

HUMAN MOLECULAR GENETICS (2012)

Article Biotechnology & Applied Microbiology

Restoration of Vision in the pde6β-deficient Dog, a Large Animal Model of Rod-cone Dystrophy

Lolita Petit et al.

MOLECULAR THERAPY (2012)

Article Multidisciplinary Sciences

AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia

Ji-Jing Pang et al.

PLOS ONE (2012)

Article Multidisciplinary Sciences

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

William A. Beltran et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

Article Biochemistry & Molecular Biology

The role of RPGR in cilia formation and actin stability

Milica Gakovic et al.

HUMAN MOLECULAR GENETICS (2011)

Article Genetics & Heredity

Allelic and Phenotypic Heterogeneity in ABCA4 Mutations

Tomas R. Burke et al.

OPHTHALMIC GENETICS (2011)

Article Ophthalmology

Whirlin Replacement Restores the Formation of the USH2 Protein Complex in Whirlin Knockout Photoreceptors

Junhuang Zou et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)

Review Ophthalmology

AAV and Compacted DNA Nanoparticles for the Treatment of Retinal Disorders: Challenges and Future Prospects

Zongchao Han et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2011)

Article Biochemistry & Molecular Biology

Gene therapy rescues cone function in congenital achromatopsia

Andras M. Komaromy et al.

HUMAN MOLECULAR GENETICS (2010)

Review Genetics & Heredity

Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait

Alan F. Wright et al.

NATURE REVIEWS GENETICS (2010)

Article Genetics & Heredity

Historical evolution in the understanding of Stargardt macular dystrophy

Gerald Allen Fishman

OPHTHALMIC GENETICS (2010)

Article Genetics & Heredity

Mutations in SPATA7 Cause Leber Congenital Amaurosis and Juvenile Retinitis Pigmentosa

Hui Wang et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2009)

Article Biochemistry & Molecular Biology

New mouse models for recessive retinitis pigmentosa caused by mutations in the Pde6a gene

Kenji Sakamoto et al.

HUMAN MOLECULAR GENETICS (2009)

Review Veterinary Sciences

AAV-Mediated Gene Therapy for Retinal Disorders in Large Animal Models

Knut Stieger et al.

ILAR JOURNAL (2009)

Article Ophthalmology

Genetic Etiology and Clinical Consequences of Complete and Incomplete Achromatopsia

Alberta A. H. J. Thiadens et al.

OPHTHALMOLOGY (2009)

Review Biochemistry & Molecular Biology

Clinical gene therapy using recombinant adeno-associated virus vectors

C. Mueller et al.

GENE THERAPY (2008)

Article Medicine, Research & Experimental

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Mariacarmela Allocca et al.

JOURNAL OF CLINICAL INVESTIGATION (2008)

Article Biotechnology & Applied Microbiology

Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediated gene transfer

Jeannette Bennicelli et al.

MOLECULAR THERAPY (2008)

Article Medicine, General & Internal

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Albert M. Maguire et al.

NEW ENGLAND JOURNAL OF MEDICINE (2008)

Letter Ophthalmology

Reflux after intravitreal injection of bevacizumab

Camiel J. F. Boon et al.

OPHTHALMOLOGY (2008)

Review Ophthalmology

Leber congenital amaurosis: Genes, proteins and disease mechanisms

Anneke I. den Hollander et al.

PROGRESS IN RETINAL AND EYE RESEARCH (2008)

Article Biochemistry & Molecular Biology

Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B

T. Hashimoto et al.

GENE THERAPY (2007)

Article Neurosciences

Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery

M. Gorbatyuk et al.

VISION RESEARCH (2007)

Article Ophthalmology

Novel phenotypic and genotypic findings in X-linked retinoschisis

Stephen H. Tsang et al.

ARCHIVES OF OPHTHALMOLOGY (2007)

Article Ophthalmology

Perspective on genes and mutations causing retinitis pigmentosa

Stephen P. Daiger et al.

ARCHIVES OF OPHTHALMOLOGY (2007)

Review Genetics & Heredity

X-linked retinoschisis: an update

Stephen K. Sikkink et al.

JOURNAL OF MEDICAL GENETICS (2007)

Review Medicine, General & Internal

Retinitis pigmentosa

Dyonne T. Hartong et al.

LANCET (2006)

Review Genetics & Heredity

Retinitis pigmentosa

Christian Hamel

ORPHANET JOURNAL OF RARE DISEASES (2006)

Article Biotechnology & Applied Microbiology

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)

Article Biotechnology & Applied Microbiology

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

Samuel G. Jacobson et al.

MOLECULAR THERAPY (2006)

Article Ophthalmology

Genotype-phenotype correlation of mouse Pde6b mutations

AW Hart et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2005)

Article Multidisciplinary Sciences

Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

SG Jacobson et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)

Review Genetics & Heredity

The genetics of inherited macular dystrophies

M Michaelides et al.

JOURNAL OF MEDICAL GENETICS (2003)

Review Physiology

Cyclic nucleotide-gated ion channels

UB Kaupp et al.

PHYSIOLOGICAL REVIEWS (2002)

Review Ophthalmology

Genetic animal models for retinal degeneration

S Fauser et al.

SURVEY OF OPHTHALMOLOGY (2002)

Review Biochemistry & Molecular Biology

Molecular genetics of Leber congenital amaurosis

FPM Cremers et al.

HUMAN MOLECULAR GENETICS (2002)