List of works by William W Hauswirth

A comprehensive review of retinal gene therapy

scientific article

AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy.

scientific article published on 16 February 2016

AAV-mediated and pharmacological induction of Hsp70 expression stimulates survival of retinal ganglion cells following axonal injury.

scientific article published on 27 November 2014

AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia

scientific article (publication date: 2012)

AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.

scientific article published on February 2013

AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.

scientific article published on 20 March 2014

AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice

scientific article

AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa

scientific article published on 12 May 2015

ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy.

scientific article published on 26 July 2011

Achromatopsia as a potential candidate for gene therapy.

scientific article

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

scientific article published on December 2015

Angiostatin overexpression is associated with an improvement in chronic kidney injury by an anti-inflammatory mechanism.

scientific article published on 29 October 2008

BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.

scientific article published on 5 March 2018

CCN2/CTGF regulates neovessel formation via targeting structurally conserved cystine knot motifs in multiple angiogenic regulators.

scientific article

CHIP, a carboxy terminus HSP-70 interacting protein, prevents cell death induced by endoplasmic reticulum stress in the central nervous system.

scientific article published on January 2014

Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat

scientific article published on 8 June 2015

Characterization of intravitreally delivered capsid mutant AAV2-Cre vector to induce tissue-specific mutations in murine retinal ganglion cells.

scientific article published on 29 July 2016

Clarin-1, encoded by the Usher Syndrome III causative gene, forms a membranous microdomain: possible role of clarin-1 in organizing the actin cytoskeleton

scientific article

Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium

scientific article published on 01 January 2018

Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma

article

Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE

scientific article

Cone Phosphodiesterase-6γ' Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits

scientific article published on 09 July 2018

Cone phosphodiesterase-6α' restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse

scientific article published on July 2013

Cone specific promoter for use in gene therapy of retinal degenerative diseases.

scientific article published on January 2014

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

scientific article published on 25 November 2015

Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice.

scientific article

DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration

scientific article

DICER1/Alu RNA dysmetabolism induces Caspase-8-mediated cell death in age-related macular degeneration

scientific article

Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature development

scientific article

Diabetic eNOS-knockout mice develop accelerated retinopathy.

scientific article

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

article

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

scientific article published on April 2014

ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability

scientific article published on 23 January 2018

ER stress is involved in T17M rhodopsin-induced retinal degeneration.

scientific article

ERK1/2 activation is a therapeutic target in age-related macular degeneration.

scientific article

Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system

scientific article

Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors.

scientific article published on 26 January 2017

Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs

scientific article

Flicker cone function in normal and day blind sheep: a large animal model for human achromatopsia caused by CNGA3 mutation

scientific article published on 10 September 2014

Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.

scientific article

Functional interchangeability of rod and cone transducin alpha-subunits.

scholarly article

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

scientific article

Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.

scientific article published on March 2017

Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.

scientific article

Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results

scientific article published on 21 June 2017

Gene Therapy for MERTK-Associated Retinal Degenerations.

scientific article published on January 2016

Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR

scientific article

Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation

scientific article

Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model.

scientific article published on January 2012

Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.

scientific article

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

scientific article

Gene therapy in the rd6 mouse model of retinal degeneration.

scientific article published on January 2014

Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model.

scientific article

Gene therapy rescues cone function in congenital achromatopsia

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

scientific article published on 23 January 2012

Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

scientific article published on 31 August 2011

Gene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis.

scientific article

Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response

scientific article published on 20 February 2015

Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.

scientific article published on 27 July 2017

Genetically targeted binary labeling of retinal neurons.

scientific article

Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex

scientific article published on 24 December 2015

Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina.

scientific article published on 20 July 2016

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

scientific article

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

scholarly article

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

scientific article published on 22 January 2013

Imaging the response of the retina to electrical stimulation with genetically encoded calcium indicators.

scientific article

Improvement and decline in vision with gene therapy in childhood blindness.

scientific article

Improvement in vision: a new goal for treatment of hereditary retinal degenerations

scientific article published on May 2015

In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses

scientific article

Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus.

scientific article

Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule ⬇️

scientific article published on October 26, 2010

Inhibitor of apoptosis-stimulating protein of p53 (iASPP) is required for neuronal survival after axonal injury.

scientific article

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.

scientific article

Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1.

scientific article published on September 2010

Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1.

scientific article

Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model

scientific article published on 31 October 2011

Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1.

scientific article published on 30 September 2017

Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.

scientific article published on 9 September 2011

Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.

scientific article published on 28 March 2012

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

scientific article published on 14 October 2005

Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

scientific article

Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic Neuropathy.

scientific article published on 3 April 2018

Lowering blood pressure blocks mesangiolysis and mesangial nodules, but not tubulointerstitial injury, in diabetic eNOS knockout mice.

scientific article published on 26 February 2009

Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation

scientific article

Modeling and Preventing Progressive Hearing Loss in Usher Syndrome III.

scientific article

Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel.

scientific article

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector ⬇️

article published in the Proceedings of the National Academy of Sciences of the United States of America

NADH-dehydrogenase type-2 suppresses irreversible visual loss and neurodegeneration in the EAE animal model of MS.

scientific article published on 11 June 2013

Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy

scientific article

Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization.

scientific article

Numb regulates the polarized delivery of cyclic nucleotide-gated ion channels in rod photoreceptor cilia

scientific article

Occludin S490 Phosphorylation Regulates Vascular Endothelial Growth Factor-Induced Retinal Neovascularization.

scientific article published on 13 July 2016

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

scientific article published on 26 May 2017

Organizational motifs for ground squirrel cone bipolar cells

scientific article

Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice

scientific article published on 01 January 2018

Overexpression of the X-Linked Inhibitor of Apoptosis Protects Against Retinal Degeneration in a Feline Model of Retinal Detachment

scientific article published on 23 March 2017

PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.

scientific article published on 10 August 2016

Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium

scientific article published on October 2015

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

scientific article published on 20 November 2017

Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

scientific article

Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1.

scientific article published on 18 June 2013

Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs

scientific article

Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs

scientific article published on 01 April 2016

Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.

scientific article published on March 2013

Pseudo-fovea formation after gene therapy for RPE65-LCA.

scientific article

RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.

scientific article published on 04 June 2013

REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.

scientific article

RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model

scientific article

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS

scientific article published on 21 June 2017

Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.

scientific article

Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs.

scientific article published on 7 April 2016

Reply

Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration.

scientific article published in May 2013

Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector

scientific article published on 28 December 2013

Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia

scientific article published on August 2016

Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78

scientific article

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.

scientific article

Retinal angiogenesis suppression through small molecule activation of p53

scientific article published on 09 September 2013

Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.

scientific article

Role of connective tissue growth factor in the retinal vasculature during development and ischemia

scientific article

STAT3 promotes survival of mutant photoreceptors in inherited photoreceptor degeneration models

scientific article

Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

scientific article published on March 2016

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

scientific article published on March 2016

Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs.

scientific article published on 12 October 2017

Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.

scientific article

Selective tropism of the recombinant adeno-associated virus 9 serotype for rat cardiac tissue.

scientific article published in January 2010

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

scientific article

Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.

scientific article

Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia

article

Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors

scientific article

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

scientific article

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

scientific article

Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector.

scientific article published on January 2012

Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.

scientific article published on 10 September 2015

Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.

scientific article published on January 2014

Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.

scientific article published on 16 January 2015

Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration

scientific article published on 13 September 2016

Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide

scientific article

The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.

scientific article

Toward a higher fidelity model of AMD

scientific article published on 01 January 2008

Towards optogenetic sensory replacement.

scientific article

Toxicology and Pharmacology of an AAV Vector Expressing Codon-optimized RPGR in RPGR-deficient Rd9 Mice

article

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

scientific article

Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

scientific article

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

scientific article

Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats.

scientific article published on 06 April 2012

Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model

scientific article

Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia

scientific article published on 8 April 2015

XIAP effects on retinal detachment-induced photoreceptor apoptosis [corrected].

scientific article published on 05 December 2008

XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina

scientific article

miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.

scientific article published on 15 June 2017

rAAV8-733-Mediated Gene Transfer of CHIP/Stub-1 Prevents Hippocampal Neuronal Death in Experimental Brain Ischemia.

scientific article published on 31 December 2016

γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines.

scientific article

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