Children’s Hospital of Philadelphia (CHOP)

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.

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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Children’s Hospital of Philadelphia (CHOP) Research

Penn Medicine and Children’s Hospital of Philadelphia to Host Symposium on Future of Cell and Gene Therapies
on April 29, 2021 at 5:24 am
PHILADELPHIA— Penn Medicine and Children's Hospital of Philadelphia (CHOP) will host a virtual event on May 6 and 7 that will bring together cell ...
Children's Hospital of Philadelphia names Boston health care executive as new CFO
on April 20, 2021 at 6:30 am
Children's Hospital of Philadelphia named a longtime health care industry executive as its new CFO. Sophia G. Holder joins CHOP from Boston Children's Hospital, where she was vice president of finance ...
Children's Hospital of Philadelphia Names New CFO
on April 19, 2021 at 12:31 pm
Children's Hospital of Philadelphia stated that Holder's role will entail making sure that the "hospital thrives in an evolving healthcare ecosystem." ...
Children's Hospital of Philadelphia Appoints Sophia G. Holder as New Executive Vice President and Chief Financial Officer
on April 19, 2021 at 10:19 am
Children's Hospital of Philadelphia (CHOP) has appointed Sophia G. Holder as Executive Vice President and Chief Financial Officer. In ...
CHOP-led research study identifies key target in treatment-resistant hemophilia A
on April 15, 2021 at 7:24 am
The research team, led by CHOP and Indiana ... 10.1172/JCI142906 About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric ...

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Dynamic changes in early childhood development may lead to changes in autism diagnosis
on April 28, 2021 at 8:55 am
Philadelphia, April 28, 2021 - Researchers from the Center for Autism Research (CAR) at Children's Hospital of Philadelphia (CHOP) found that difficulties in diagnosing toddlers with autism ...
Immune tolerance in hemophilia can be modulated by B cell activating factor
on April 19, 2021 at 9:50 pm
A group of scientists have just made a key discovery that could prevent ... at Indiana University School of Medicine, Children's Hospital of Philadelphia and University of Pennsylvania joined ...
B cell activating factor possible key to hemophilia immune tolerance
on April 18, 2021 at 9:00 pm
A group of scientists have just made a key discovery that could prevent and eradicate immune responses that lead to treatment failure in about one-third of people with severe hemophilia A.
B cell activating factor possible key to hemophilia immune tolerance
on April 18, 2021 at 5:00 pm
A group of scientists have just made a key discovery that could prevent ... at Indiana University School of Medicine, Children's Hospital of Philadelphia and University of Pennsylvania joined ...
Children's Hospital of Philadelphia Opens New Specialty Care Center in Abington
on April 12, 2021 at 6:26 am
PHILADELPHIA, April 12, 2021 /PRNewswire/ -- Children's Hospital of Philadelphia (CHOP) today announced the opening of a new, 37,000-square-foot Specialty Care Center in Abington, PA. This is the ...