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Cool Videos: Myotonic Dystrophy
Today, I’d like to share a video that tells the inspirational story of two young Massachusetts Institute of Technology (MIT) researchers who are taking aim at a genetic disease that has touched both...
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Snapshots of Life: Lost Connections in Pompe Disease
Caption: Abnormal connections between leg muscle fibers (red) and nerves (green) in Pompe disease.Credit: Darin J. Falk, A. Gary Todd, Robin Yoon, and Barry J. Byrne, University of Florida, Gainesville...
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Cool Videos: Patching and Sealing the Cell Membrane
Bill Bement describes himself as a guy who “passionately, obsessively, and almost feverishly” loves to study cells. His excitement comes through in our final installment of the American Society for...
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Creative Minds: Can Diseased Cells Help to Make Their Own Drugs?
Matthew Disney Matthew Disney grew up in a large family in Baltimore in the 1980s. While his mother worked nights, Disney and his younger brother often tagged along with their father in these...
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Huntington’s Disease: Gene Editing Shows Promise in Mouse Studies
My father was a folk song collector, and I grew up listening to the music of Woody Guthrie. On July 14th, folk music enthusiasts will be celebrating the 105th anniversary of Guthrie’s birth in his...
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Robotic Exoskeleton Could Be Right Step Forward for Kids with Cerebral Palsy
More than 17 million people around the world are living with cerebral palsy, a movement disorder that occurs when motor areas of a child’s brain do not develop correctly or are damaged early in life....
View ArticleSharing a Story of Hope
Whether by snail mail, email, or social media, it’s the time of year for catching up with family and friends. As NIH Director, I’m also fortunate to hear from some of the amazing people who’ve been...
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Gene Editing in Dogs Boosts Hope for Kids with Muscular Dystrophy
Caption: A CRISPR/cas9 gene editing-based treatment restored production of dystrophin proteins (green) in the diaphragm muscles of dogs with Duchenne muscular dystrophy.Credit: UT Southwestern CRISPR...
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More Progress Toward Gene Editing for Kids with Muscular Dystrophy
Caption: Muscles of untreated mouse model of Duchenne muscular dystrophy (left) compared to muscles of similar mice one year after gene-editing treatment (right). Dystrophin production (green) is...
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Nano-Sized Solution for Efficient and Versatile CRISPR Gene Editing
Caption: Nanocapsule containing its gene-editing payload. Credit: Guojun Chen and Amr Abdeen, University of Wisconsin-Madison If used to make non-heritable genetic changes, CRISPR gene-editing...
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Gene-Editing Advance Puts More Gene-Based Cures Within Reach
Caption: The prime editing system (left) contains three parts: two enzymes, Cas9 and reverse transcriptase, and an engineered guide RNA, pegRNA. Unlike regular CRISPR gene editing, prime editing nicks...
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Experts Conclude Heritable Human Genome Editing Not Ready for Clinical...
We stand at a critical juncture in the history of science. CRISPR and other innovative genome editing systems have given researchers the ability to make very precise changes in the sequence, or...
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Millions of Single-Cell Analyses Yield Most Comprehensive Human Cell Atlas Yet
There are 37 trillion or so cells in our bodies that work together to give us life. But it may surprise you that we still haven’t put a good number on how many distinct cell types there are within...
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