2013 - The Llura Liggett Gund Award, Foundation Fighting Blindness for using non-harmful adeno-associated viruses (AAVs) to transport healthy DNA into retinal cells at the back of the eye, showing that gene therapy could restore vision in animal models, including Briard dogs. His team’s approach was then used in human clinical trials where children and young adults virtually blind from Leber congenital amaurosis (LCA) have had significant vision restored.
His main research concerns Retina, Molecular biology, Retinal, Genetics and Genetic enhancement. His studies deal with areas such as Ophthalmology, Neurotrophic factors and Pathology as well as Retina. The study incorporates disciplines such as Mutant, Gene, Recombinant DNA, Green fluorescent protein and DNA in addition to Molecular biology.
His is doing research in Retinal degeneration, RPE65, Gene therapy of the human retina, Retinal pigment epithelium and Leber's congenital amaurosis, both of which are found in Retinal. His biological study deals with issues like Retinopathy, which deal with fields such as Diabetic retinopathy. His Genetic enhancement study combines topics in areas such as Disease and Bioinformatics.
The scientist’s investigation covers issues in Cell biology, Genetic enhancement, Molecular biology, Retina and Retinal. His Cell biology research includes elements of Knockout mouse and Gene expression. As part of the same scientific family, William W. Hauswirth usually focuses on Genetic enhancement, concentrating on Virology and intersecting with Transduction and Recombinant DNA.
As a member of one scientific family, William W. Hauswirth mostly works in the field of Molecular biology, focusing on Rhodopsin and, on occasion, Ribozyme. His study focuses on the intersection of Retina and fields such as Optic nerve with connections in the field of Retinal ganglion. His Retinal study is associated with Ophthalmology.
William W. Hauswirth spends much of his time researching Genetic enhancement, Cell biology, Retina, Retinal and Ophthalmology. His work deals with themes such as Transgene, Virology, Vector, Mutant and Molecular biology, which intersect with Genetic enhancement. William W. Hauswirth regularly ties together related areas like Gene delivery in his Molecular biology studies.
His work on Function as part of general Cell biology research is frequently linked to Cone, thereby connecting diverse disciplines of science. The concepts of his Retina study are interwoven with issues in Cell type, Optic nerve and Pathology. His study in Retinal degeneration and Retinitis pigmentosa is carried out as part of his studies in Retinal.
His scientific interests lie mostly in Genetic enhancement, Retinal, Ophthalmology, Electroretinography and Molecular biology. His Genetic enhancement research is multidisciplinary, incorporating perspectives in Vector, Mutant and Transgene. As a part of the same scientific study, William W. Hauswirth usually deals with the Retinal, concentrating on Retina and frequently concerns with Pathology.
His work carried out in the field of Electroretinography brings together such families of science as Color vision and Achromatopsia. He interconnects GTPase, RAB11A, Xenopus, Protein subcellular localization prediction and Zebrafish in the investigation of issues within Molecular biology. He studied Gene and Cell biology that intersect with Vascular permeability and Transmembrane protein.
This overview was generated by a machine learning system which analysed the scientist’s body of work. If you have any feedback, you can contact us here.
Gene therapy restores vision in a canine model of childhood blindness.
Gregory M. Acland;Gustavo D. Aguirre;Jharna Ray;Qi Zhang.
Nature Genetics (2001)
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
William W. Hauswirth;Tomas S. Aleman;Shalesh Kaushal;Artur V. Cideciyan.
Human Gene Therapy (2008)
A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells.
Sergei Zolotukhin;Mark Potter;William W. Hauswirth;John Guy.
Journal of Virology (1996)
A muscleblind knockout model for myotonic dystrophy.
Rahul N. Kanadia;Karen A. Johnstone;Ami Mankodi;Codrin Lungu.
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
Artur V. Cideciyan;Tomas S. Aleman;Sanford L. Boye;Sharon B. Schwartz.
Proceedings of the National Academy of Sciences of the United States of America (2008)
Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
Samuel G. Jacobson;Artur V. Cideciyan;Ramakrishna Ratnakaram;Elise Heon.
Archives of Ophthalmology (2012)
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors.
Sergei Zolotukhin;Mark Potter;Irene Zolotukhin;Yoshihisa Sakai.
DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration
Hiroki Kaneko;Sami Dridi;Valeria Tarallo;Bradley D. Gelfand.
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
Gregory M. Acland;Gustavo D. Aguirre;Jean Bennett;Tomas S. Aleman.
Molecular Therapy (2005)
Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.
Alfred S. Lewin;Kimberly A. Drenser;William W. Hauswirth;Shimpei Nishikawa.
Nature Medicine (1998)
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