Tag Archive: Raymond G. Perelman Center for Cellular and Molecular Therapeutics

Nov 6 2018

Realizing the Promise of Gene Therapy: Q&A With Clinical Vector Core Directors

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Oct 8 2018

The Science Behind Gene Therapy: Creating Breakthroughs One Gene at a Time

For more than 20 years, researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania have been at the forefront of taking a system perfected by nature — a virus — and transforming it into breakthrough gene therapies for rare single-gene diseases. CHOP was the first pediatric research institution to develop chimeric antigen receptor T cell (CAR-T) therapy for acute lymphoblastic leukemia. In this approach, viral vectors are used to modify a patient’s own T cells, training them to track down and eliminate the circulating cancer cells.

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May 1 2018

Does an Impaired Biological Pathway Help Explain Heart Problems in Huntington’s Disease?

The Findings:

Researchers gained new insights into the heart problems that are the second leading cause of death in patients with Huntington’s disease (HD). An incurable, inherited disease with progressive loss of brain cells and motor function, HD occurs when a defective gene produces repeated copies of a protein called huntingtin, or HTT. The mutant HTT (mHTT) protein disrupts multiple fundamental cellular processes along the mTORC1 pathway that promotes cell growth and metabolism. The study team described how decreased mTORC1 activity contributed to the development of heart disease with stress in mouse models of HD. By restoring cardiac mTORC1 activity, the researchers improved the animals’ heart function and survival over the course of the study.

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Dec 21 2017

Groundbreaking Gene Therapy Approved for Inherited Retinal Blindness

More than a decade of studies led by researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania culminated this week with the U.S. Food and Drug Administration’s approval of an innovative one-time gene therapy product indicated for the treatment of patients with a rare, inherited form of retinal blindness.

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Jul 29 2016

CHOP Research In the News: New Rare Disease, FA Woodstock, Bioengineering, and Protecting Toddlers from Child Abuse

CHOP Research In the NewsOne of the things that is so exciting about research at CHOP is that our researchers are working to improve the health and lives of children in such a broad range of ways.

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