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A new gene therapy transplantation technique could improve treatment of neurodegenerative disease

December 06, 2017
Alessandra Biffi, MD, led a study that shows the efficacy of a new gene therapy transplantation technique that could have future applications for treating neurodegenerative diseases.

New gene therapy clinical trial offers hope for sickle cell disease

November 14, 2017
David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.

Two Dana-Farber/Boston Children's faculty members elected to National Academy of Medicine

October 16, 2017
Two Dana-Farber/Boston Children's Cancer and Blood Disorders Center faculty members, Scott Allen Armstrong, MD, PhD, and Scott Pomeroy, MD, PhD, have been elected to the National Academy of Medicine (NAM).

The space between heartache and happiness: Two sons with adrenoleukodystrophy and a gene therapy clinical trial

October 04, 2017
For the Rojas family, a clinical trial of gene therapy for adrenoleukodystrophy has meant both happiness and heartache.

Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial

October 04, 2017
The devastating neurodegenerative disease typically affects young boys and causes death within 10 years

Dana-Farber/Boston Children's responds to FDA approval of CAR-T cell therapy

August 30, 2017
The FDA's approval of CAR-T therapy for refractory pediatric and young adult ALL underscores the potential of CAR T-cell therapies, a form of cancer immunotherapy, to treat a wide range of cancers, researchers say.

Vietnamese boy thrives after gene therapy for Wiskott-Aldrich syndrome

August 23, 2017
After being diagnosed with Wiskott-Aldrich syndrome, Duy Anh "Harry" Le traveled with his family to Boston for a gene therapy clinical trial, and is now a happy and healthy two-year-old.

Novel CRISPR-Cas9 genetic screening approach enables discovery of new drug targets to aid cancer immunotherapy

July 19, 2017
A novel screening method, using CRISPR-Cas9 genome editing technology to test the function of thousands of tumor genes in mice, has revealed new drug targets that could potentially enhance the effectiveness of PD-1 checkpoint inhibitors, a promising new class of cancer immunotherapy.

Dana-Farber/Boston Children's again named a top national pediatric cancer program

June 27, 2017
U.S. News & World Report has ranked Dana-Farber/Boston Children's Cancer and Blood Disorders Center the nation's #2 pediatric cancer program in its new 2017-18 Best Children's Hospitals report.

Gene Therapy 2.0

March 15, 2017
Levi Looks, diagnosed through newborn screening with “bubble boy” disease, is a healthy toddler thanks to gene therapy, resurgent now after early setbacks, MIT Technology Review reports.
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