Tag Archive: Gene therapy

Aug 15 2014

Forced Looping Viable Strategy to Reverse Globin Switch

Investigators at The Children’s Hospital of Philadelphia are exploring a new gene therapy approach that aims to reactivate the production of fetal hemoglobin as a potential intervention for patients with sickle cell disease.

Permanent link to this article: http://www.research.chop.edu/blog/forced-looping-viable-strategy-reverse-globin-switch/

Apr 14 2014

Forbes Article Features CHOP Gene Therapy Expert

A recent Forbes article on the resurrection of the gene therapy business featured The Children’s Hospital of Philadelphia gene therapy expert Katherine A. High, MD.

Permanent link to this article: http://www.research.chop.edu/blog/forbes-article-features-chop-gene-therapy-expert/

Apr 02 2014

Prominent Gene Therapy Expert Joins CHOP

Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1.

Permanent link to this article: http://www.research.chop.edu/blog/prominent-gene-therapy-expert-joins-chop/

Jan 16 2014

Repairing DNA, One Cell at a Time

High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA.

Permanent link to this article: http://www.research.chop.edu/blog/repairing-dna-one-cell-at-a-time/

Dec 11 2013

CHOP Hematologist and Gene Therapy Researcher Honored

For her pioneering hemophilia and gene therapy research, Children’s Hospital hematologist Katherine A. High, MD, director of the Center for Cellular and Molecular Therapeutics (CCMT), was recently honored with the 2013 E. Donnall Thomas Lecture and Prize at the American Society of Hematology (ASH) Annual Meeting.

Permanent link to this article: http://www.research.chop.edu/blog/chop-hematologist-gene-therapy-researcher-honored/

Jul 25 2013

Children’s Hospital Researchers Develop Bioengineered Decoy to Improve Gene Therapy

Gene therapy researchers at The Children’s Hospital of Philadelphia have produced a bioengineered decoy that by fooling the immune system prevents it from undermining the benefits delivered by a corrective gene.

Permanent link to this article: http://www.research.chop.edu/blog/childrens-hospital-researchers-develop-bioengineered-decoy-to-improve-gene-therapy/

May 02 2013

CHOP, Penn Gene Therapy Trial for Blindness Honored

A groundbreaking clinical trial of gene therapy for a form of congenital blindness, sponsored by The Children’s Hospital of Philadelphia in collaboration with Penn Medicine, was recently recognized with the Distinguished Clinical Research Achievement Award from the Clinical Research Forum, an organization of clinical research centers, industry, and volunteer groups.

Permanent link to this article: http://www.research.chop.edu/blog/chop-penn-gene-therapy-trial-for-blindness-honored/

Dec 28 2012

Gene Therapy Shows Promise For Helping Children With Canavan Disease

Canavan disease is a rare inherited neurological disorder with devastating effect. The lack of a specific enzyme, called aspartoacylase, causes the body’s central nervous system to break down. The disease is usually fatal before a child reaches the teenage years.

Permanent link to this article: http://www.research.chop.edu/blog/gene-therapy-shows-promise-for-helping-children-with-canavan-disease/

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