Gene therapy’s new hope: A neuron-targeting virus is saving infant lives

November 01, 2017

CENTREVILLE, VIRGINIA—Nothing unusual jumps out upon meeting Evelyn, a bubbly almost-3-year-old with red curls—except that she should not be here, chatting with a visitor in her family’s living room, twirling in her tights to the Pharrell Williams song “Happy.”

Penn Scientists Use CRISPR for First Time to Correct Clotting in Newborn and Adult Mice

November 30, 2016

Penn Scientists Use CRISPR for First Time to Correct Clotting in Newborn and Adult Mice


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Gene Therapy: Industrial Strength

September 08, 2016

Gene Therapy: Industrial Strength


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Penn Medicine to Develop the Next Generation of Viral Vectors — called AAV 3.0™ — for Gene Therapies and Genome Editing

June 29, 2016

Penn Medicine to Develop the Next Generation of Viral Vectors — called AAV 3.0™ — for Gene Therapies and Genome Editing


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June 28, 2016

Penn Orphan Disease Center Names Director of Neurogenetics and Grant Program Awardees for Rare Genetic Disorder


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February 05, 2016

Penn Medicine and LouLou Foundation Create Program of Excellence for Rare Genetic Disorder that Affects Children


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Penn Alumna and Employee Fights Valiantly Against Rare Form of Disease

February 02, 2016

Penn Alumna and Employee Fights Valiantly Against Rare Form of Disease


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Penn Medicine Researcher Receives Champion of Hope Award

October 01, 2015

Penn Medicine Researcher Receives Champion of Hope Award


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August 14, 2015

FDA Issues Guidance on Rare Disease Drug Development


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