Tag Archive: Gene therapy

Dec 16 2016

CHOP Research In the News: Global Trial of CTL019, Gene Therapy for Hemophilia, Child Abuse Within Military, New Radiologist-in-Chief, ADHD in Preschool

Your holiday season has been hectic, no doubt. Catch up with an early gift from us: Our biweekly roundup of research news from Children’s Hospital of Philadelphia comes with all the trimmings!

Permanent link to this article: http://blog.research.chop.edu/chop-research-news-global-trial-ctl019-gene-therapy-hemophilia-child-abuse-within-military-new-radiologist-chief-adhd-preschool/

Oct 21 2016

CHOP Research In the News: Cancer Moonshot Yields Data-Sharing Initiatives, Hemophilia Surprise, Mitochondrial Medal

Buckle your seatbelts because this has been a busy week for research news at Children’s Hospital of Philadelphia.

Permanent link to this article: http://blog.research.chop.edu/chop-research-news-cancer-moonshot-yields-data-sharing-initiatives-hemophilia-surprise-mitochondrial-medal/

Jan 25 2016

CHOP Research In the News

Research at The Children’s Hospital of Philadelphia never stops. To help keep you informed about new discoveries and CHOP researchers’ views on timely topics, we are bringing together a roundup of news highlights. Look for this as a recurring feature here on Cornerstone.

Permanent link to this article: http://blog.research.chop.edu/chop-research-news-this-week-cancer-moonshot-autism-genes-gene-transfer/

Nov 12 2015

Targeted Gene Therapy Aimed at Devastating Childhood Disease

When something important is missing, we often search for a replacement. After many years of looking, a team of researchers at The Children’s Hospital of Philadelphia and the University of Missouri have found a way to substitute for a missing gene linked to a relentless childhood neurodegenerative disease.

Permanent link to this article: http://blog.research.chop.edu/targeted-gene-therapy-aimed-at-devastating-childhood-disease/

Apr 22 2015

Investigation Bolsters Use of Variant Protein in Hemophilia Gene Therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated animals with the bleeding disorder hemophilia.

Permanent link to this article: http://blog.research.chop.edu/investigation-bolsters-use-of-variant-protein-in-hemophilia-gene-therapy/

Jan 12 2015

Researchers Lay Groundwork for Novel Therapy for Huntington’s Disease

Researchers in the Center for Cellular and Molecular Therapeutics (CCMT) at The Children’s Hospital of Philadelphia suggest that an intricate pathway crucial to the development of Huntington’s disease (HD) rests on a “biological teeter-totter” that when carefully balanced could help to control this devastating neurodegenerative disorder.

Permanent link to this article: http://blog.research.chop.edu/researchers-lay-groundwork-novel-therapy-huntingtons-disease/

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://blog.research.chop.edu/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://blog.research.chop.edu/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://blog.research.chop.edu/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://blog.research.chop.edu/repairing-dna-one-cell-at-a-time/

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