New gene therapy corrects a form of inherited macular degeneration in canine model

(University of Pennsylvania) Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable.
Source: EurekAlert! - Biology - Category: Biology Source Type: news

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Bannockburn, Illinois-based gene therapy company AveXis is bringing 200 new jobs to Durham through a new $55 million manufacturing center, Gov. Roy Cooper ’s office announced Tuesday. The clinical-stage company is focused on developing therapies for the treatment of rare genetic diseases in the field of neurology. Novartis completed its acquisition of AveXis earlier this month. “Life science companies understand the many advantages our state off ers manufacturers, particularly the investments…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
Contrast developer Bracco Imaging is collaborating with longtime partner Shanghai...Read more on AuntMinnie.comRelated Reading: Bracco hits Jubilant with patent complaint on rubidium PET Bracco lands new FDA approval for MultiHance Bracco targets gene therapy for ultrasound contrast Bracco declines comment on Norris gadolinium lawsuit Bracco Imaging acquires SurgVision
Source: AuntMinnie.com Headlines - Category: Radiology Source Type: news
Bannockburn, Illinois-based gene therapy company AveXis is bringing 200 new jobs to Durham through a new $55 million manufacturing center, Gov. Roy Cooper ’s office announced Tuesday. The clinical-stage company is focused on developing therapies for the treatment of rare genetic diseases in the field of neurology. Novartis completed its acquisition of AveXis earlier this month. “Life science companies understand the many advantages our state off ers manufacturers, particularly the investments…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
Spark Therapeutcis and Pfizer have cleared a key hurdle in their effort to develop a gene therapy treatment for patient with hemophilia B. On Tuesday, the companies said all 15 hemophilia B patients in Philadelphia-based Spark’s ongoing phase-I/II clinical trial of its investigational gene therapy SPK-9001 had discontinued routine infusions of factor IX concentrates – and none of the 15 participants experienced serious adverse ev ents. Hemophilia B is a rare, genetic bleeding disorder found…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
The long-term success of prosthetic vascular grafts for revascularization in peripheral arterial disease is limited by the development of anastomotic neointimal hyperplasia (ANIH). We have constructed a bioactive prosthetic graft material (BPGM) by coating polyethylene terephthalate with an antithrombogenic cryogel polymer layer capable of delivering biologic agents to human aortic endothelial cells in vitro. Our goal is to evaluate our hybrid prosthetic graft material in vivo using a rabbit carotid interposition bypass model.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain, Published online: 22 May 2018; doi:10.1038/s41434-018-0017-8Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Date: Monday, 08 20, 2018; Speaker: Christopher P. Austin, MD, Director , NCATS/NIH; Wilson Bryan, MD, Director, Office of Tissues&Advanced Therapies, CBER/FDA; Anne Pariser, MD, Director, Office of Rare Diseases Research, NCATS/NIH; Peter Marks, MD, Director, Center for Biologics Evaluation and Research, FDA; Building 10; Masur Auditorium; Videocast Event
Source: NIH Calendar of Events - Category: American Health Source Type: events
(Mary Ann Liebert, Inc./Genetic Engineering News) Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, Memorial Sloan-Kettering Cancer Center in New York City, offers a fascinating perspective on the re-markable progress being made in the field of chimeric antigen receptor (CAR) engineered T-cell therapies to treat cancer.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Haemophilia, EarlyView.
Source: Haemophilia - Category: Hematology Authors: Source Type: research
Abstract The targeting efficiency of knockin sequences via homologous recombination (HR) is generally low. Here we describe a method we call Tild-CRISPR (targeted integration with linearized dsDNA-CRISPR), a targeting strategy in which a PCR-amplified or precisely enzyme-cut transgene donor with 800-bp homology arms is injected with Cas9 mRNA and single guide RNA into mouse zygotes. Compared with existing targeting strategies, this method achieved much higher knockin efficiency in mouse embryos, as well as brain tissue. Importantly, the Tild-CRISPR method also yielded up to 12-fold higher knockin efficiency t...
Source: Developmental Cell - Category: Cytology Authors: Tags: Dev Cell Source Type: research
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