In an article published today in the New York Times, PRF Medical Director Dr. Leslie Gordon and colleagues shared the phenomenal story of scientific collaborations that led to recent breakthroughs in genetic editing in Progeria.
Partnerships with PRF’s long-time friends and colleagues, former NIH Director Dr. Francis Collins, and gene editing expert Dr. David Liu of Harvard/MIT, have yielded extraordinary findings previously published in the journal Природа, on the potentially curative powers of base editing.
Get the full story here: A Disease That Makes Children Age Rapidly Gets Closer to a Cure
The new types of gene editing are “potentially the answer to a dream we all want to come true,”
Dr. Francis Collins
Check out some of the wonderful reader feedback:
“DNA editing splicing and re-writing technology… just a jaw dropping read. Wow…”
“For a better understanding of this disease, watch the movie “Life According to Sam” on HBO. You will be amazed by the devotion to discovering a cure for progeria by Sam’s parents. You will be amazed by Sam, whom I was fortunate to get to know while he explored playing timpani in symphony orchestra during summer music camp. He was an engaged, inquisitive multi-talented student who accomplished so much in his too short life.”
“Inspiring article! Thank you Times reporter, for directing our attention to the fights worth fighting! The good fights! Let’s declare war on disease and disorder instead of human against human. Pour money into killing viruses not each other. Please continue to make us more aware of all the good fights that are being fought in laboratories and hospitals. We need it! Thank you!”
“So moving to read. Dr. Collins is an American hero. These scientists on the cusp here deserve our funding and our admiration. The patients most of all deserve our support. What a brave bunch of people to live with this debilitating disease. Hoping for a huge breakthrough soon.”
“Loved this article! Thank you NYT. How great to read about something so promising that will help so many. Bravo to Dr. Collin’s and colleagues.”
“What a wonderful article and what an American treasure Francis Collins and David Liu are. So grateful for their selfless dedication to these rare genetic diseases.”
“Absolutely fascinating. I hope they get the approval for the clinical trial soon. Great work.”
“This is just phenomenal. I was very interested in medicine as a kid and I watched documentaries about all sorts of rare diseases. Progeria always stuck out to me. I’m elated to see this kind of work being done. I hope this is successful in curing the children who have this condition and it can be used to cure many more.”
“In the 1990s, I was a science writer for a mammalian genetics lab. Back then, the words “in vivo base editing” would have made me fall out of my chair with a joyful shout! I share Dr. Collins’ “WOW,” and I am so grateful for the clarity and optimism of this article.”
“Thank you to the author for covering this very important scientific development. Huge kudos to Dr. Collins and everyone at PRF for their tireless work. I was fortunate and humbled to have been there when PRF began and I gladly volunteered for many of their fundraising initiatives to help support their incredible efforts. I was lucky to have met Sam several times – he was such a terrific person. Seeing the success all of this hard work has accomplished and is continuing to accomplish is a true joy.”
“Dr. Collins is the kind of scientist we need. Not looking to cash out by making millions on a drug treatment, but happy to live on his NIH salary while doing the critical research that will save lives. More like him, please!”
“This is wonderful and hopeful. I was fortunate enough to know Sam- and his parents. I still think about him- his courage, tenacity, and amazing spirit. He is cheering you on from where his journey has taken him for sure!”
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