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Children’s Hospital of Philadelphia (CHOP)

July 18, 2019

The Children’s Hospital of Philadelphia (CHOP) is a children’s hospital in Philadelphia, Pennsylvania, with its primary campus located in the University City neighborhood of West Philadelphia next to the campus of the University of Pennsylvania.

A COVID-19 breathalyzer for kids?

A COVID-19 breathalyzer for kids?

A new wave of gene therapies becomes possible with the development of a “dimmer switch”

A new wave of gene therapies becomes possible with the development of a “dimmer switch”

Imagine using gene therapy to correct lethal lung diseases before death

Using CRISPR gene editing, a team from Children’s Hospital of Philadelphia (CHOP) and Penn Medicine have thwarted a lethal lung disease in an animal model in which a harmful mutation causes death within hours after birth. This proof-of-concept study, published today in Science Translational Medicine, showed that in uteroediting could be a promising new approach for treating lung diseases

Imagine using gene therapy to correct lethal lung diseases before death

Proof-of-concept in treating congenital disease before birth using CRISPR pre-natal gene editing

CHOP/Penn Medicine Team Shows first example of base-editing tool to treat a disease in utero in animal models For the first time, scientists have performed prenatal gene editing to prevent a lethal metabolic disorder in laboratory animals, offering the potential to treat human congenital diseases before birth. Published today in Nature Medicine, research from Children’s Hospital of Philadelphia (CHOP) and the Perelman

Proof-of-concept in treating congenital disease before birth using CRISPR pre-natal gene editing

What will emerging genetic tools for mitochondrial DNA replacement mean for patients?

Social and ethical issues arise beyond potential for preventing rare complex diseases Sophisticated prenatal techniques, not yet in clinical practice, offer the potential to prevent a cruel multi-system genetic disease passing from mother to child long before birth. But these emerging tools raise important questions: might there be unintended, even irreversible risks to the resulting

What will emerging genetic tools for mitochondrial DNA replacement mean for patients?

Vision-Restoring Gene Therapy Also Strengthens Visual Processing Pathways in Brain

Surprisingly rapid regrowth of unused brain connections after decades of near blindness Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children’s Hospital of

Vision-Restoring Gene Therapy Also Strengthens Visual Processing Pathways in Brain

T-Cell Therapy Puts Leukemia Patients in Extended Remission

An experimental therapy has brought prolonged remissions to a high proportion of patients who were facing death from advanced leukemia after standard treatments had failed, researchers are reporting. The therapy involves genetically programming cells from the patient’s own immune system to fight the disease. The research included 30 patients: five adults ages 26 to 60,

T-Cell Therapy Puts Leukemia Patients in Extended Remission

New gene delivery method: magnetic nanoparticles

New research in The FASEB Journal suggests that with magnetic guidance, specially designed nanoparticles can help deliver genes to injured arteries, using stents as the delivery platform Stent angioplasty saves lives, but there often are side effects and complications related to the procedure, such as arterial restenosis and thrombosis. In the June 2013 issue of The

New gene delivery method: magnetic nanoparticles

UAB researchers cure type 1 diabetes in dogs

This had never before been achieved with any other innovative therapies for diabetes. Researchers from the Universitat Autònoma de Barcelona (UAB), led by Fàtima Bosch, have shown for the first time that it is possible to cure diabetes in large animals with a single session of gene therapy. As published this week in Diabetes, the principal journal for

UAB researchers cure type 1 diabetes in dogs

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