Broad Institute of MIT and Harvard’s Post

‘𝗘𝗽𝗶𝗴𝗲𝗻𝗼𝗺𝗲 𝗲𝗱𝗶𝘁𝗼𝗿’ 𝘀𝗶𝗹𝗲𝗻𝗰𝗲𝘀 𝗴𝗲𝗻𝗲 𝘁𝗵𝗮𝘁 𝗰𝗮𝘂𝘀𝗲𝘀 𝗱𝗲𝗮𝗱𝗹𝘆 𝗯𝗿𝗮𝗶𝗻 𝗱𝗶𝘀𝗼𝗿𝗱𝗲𝗿𝘀. 𝗣𝗿𝗶𝗼𝗻 𝗱𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗮𝗿𝗲 𝗰𝗮𝘂𝘀𝗲𝗱 𝗯𝘆 𝗺𝗶𝘀𝗳𝗼𝗹𝗱𝗲𝗱 𝗽𝗿𝗼𝘁𝗲𝗶𝗻𝘀, 𝗯𝘂𝘁 𝗮 𝗻𝗲𝘄 𝘁𝗼𝗼𝗹 𝗰𝗮𝗻 𝘀𝘁𝗼𝗽 𝘁𝗵𝗲𝗺 𝗳𝗼𝗿𝗺𝗶𝗻𝗴 𝗶𝗻 𝗺𝗶𝗰𝗲. Another study showed ‘Epigenetic’ editing cuts cholesterol in mice. https://lnkd.in/dxtdTqHb A novel molecular-editing tool, CHARM (=Coupled Histone tail for Autoinhibition Release of Methyltransferase), can be delivered to the brain to help prevent #Prion disease (=Creutzfeldt–Jakob disease). This tool works by modifying the epigenome, to regulate gene activity. In mice, CHARM was found to effectively silence the gene responsible for producing disease-causing proteins in most neurons across the brain without altering the gene sequence. This development seems a significant first step towards creating a single and effective treatment for reducing harmful proteins associated with prion disease. Alice Anane #Creutzfeldt–Jakob disease, #CJD

"A significant trend in the market revolves around the rising need for personalized medicine, where targeted therapies are developed according to an individual patient’s traits. Another area experiencing growth is the utilization of APIs in gene therapy, which has demonstrated encouraging outcomes in addressing genetic disorders.” It’s so exciting to see new developments in gene therapy! #genetherapy #oligonucleotide #mrna #rna https://lnkd.in/gMXXxUJr

The gene-editing approach uses prime editors and evolved enzymes called recombinases, and could pave the way to effective one-size-fits-all gene therapies for diseases such as cystic fibrosis. https://lnkd.in/dmtWJihW

Gene therapies are under threat. Really? After learning for >30 years and spectacular success in immunodeficiences, HIV and cancers, we no longer have to prove they are life changing! Next-step is to make bioproduction easier & cheaper to promote patient access to these treatments! Together start-ups, biotech, Big pharma, regulatory agences and medical doctors are working hand to hand to make it happen. Investors just need to believe in us…

Gene therapy is the future of medicine! Researchers are altering genes to cure diseases like AIDS, cancer, and diabetes. The FDA approved several gene therapy products for rare disorders in 2023-2024, marking significant progress. Patients like Delilah, who underwent gene therapy for a rare disorder, are experiencing life-changing results. https://lnkd.in/gTQmQhRs #bioviva #genetherapy #lizparrish #biohacking #biohacker #CRISPR #biohack #fitness #healthylifestyle #motivation #keto #nootropics #nutrition #biohackers #biohackingsecrets #longevity #meditation #brainhealth #mindfulness #healthy #functionalmedicine #antiaging #heartdisease #telomeres #klotho #healthnews #dementia #alzheimers #genetherapy

✨ [RARE DISEASE DAY]   🌏 Globally more than 300 million people, most of them children, are living with one of 7000 rare diseases. Of these 7000 rare diseases, do you know that 80% are genetic diseases and nearly 95% have no #treatments?    🧬 At Genethon, more than 200 of our #scientists and #experts are dedicated from #research to clinical trials to design and develop gene therapy treatments to cure patients with rare diseases. Although gene therapy has proved its worth, winning initial victories, access to these therapeutic innovations remains an obstacle.   🔬 On the occasion of #RareDiseaseDay, we are highlighting our different #strategies to bring gene therapies to patients suffering from rare diseases such as Limb Girdle Muscular Dystrophies (LGMD), #Crigler Najjar syndrome and Duchenne muscular dystrophy. Find out more 👉 https://urlz.fr/pIDw AFM-Téléthon Rare Disease Day #genethon #RareDiseaseDay2024 #development #strategies #rarediseaseday #duchenne #LGMD #JIMR

Read this article about a new Phase 1/2 clinical trial has lead to discoveries on how a one-time gene therapy targets the genetic source leading to Huntington's disease, a devastating neurodegenerative disease.   Click below to find out more about how the discovery is made and exactly how it works… Fascinating! 🧠 #genetics #genetharapy #RNA #neurons https://lnkd.in/enZMdcSP

Rare diseases need gene therapies too. This article shows how we could get there. #genetherapy #celltherapy #biotechnology #science https://lnkd.in/gJB-38va

My BioCentury Inc. read this week highlights the exciting advancements in gene therapy from the recent ASGCT meeting! 🚀 The potential of vectorized antibodies showcased at ASGCT could extend gene therapy beyond rare genetic diseases, revolutionizing treatments for cancers, infectious diseases, and chronic inflammatory conditions. Harnessing viral vectors to deliver therapeutic antibodies is paving the way for precision medicine, opening new frontiers in healthcare. This work underscores the versatility and promise of gene therapy in addressing a broader range of health challenges. Read more about these groundbreaking developments in BioCentury's article here. #GeneTherapy #Biotechnology #HealthcareInnovation #ASGCT2024

At Prevail Therapeutics, we are dedicated to providing new options for individuals with neurodegenerative disorders and related rare diseases. We develop potentially disease-modifying gene therapies that target the root genetic causes of conditions, including Parkinson’s disease, frontotemporal dementia, and Gaucher disease. Our approach involves selecting patient populations with specific genetic mutations that we believe can be treated by introducing a healthy copy of a missing or deficient gene. Our gene therapies use adeno-associated virus (AAV) vectors, small non-replicating viruses that act as shuttles to deliver engineered DNA to human cells. Each of our gene therapy candidates is intended to be a one-time treatment to correct the key underlying genetic mutation that we believe drives disease progression. To learn more about gene therapy, visit: https://bit.ly/45Myble #GeneTherapy #NeurodegenerativeDiseases #ParkinsonsDisease #FrontotemporalDementia #GaucherDisease