The hottest Gene Therapy Substack posts right now

And their main takeaways
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Top Technology Topics

Technology indistinguishable from magic

Wrong Side of History • 470 implied HN points • 31 Dec 24
🏥 Health & Wellness Pharmaceuticals Gene Therapy Chronic Illness Public Health
  1. Medical technology has advanced a lot recently. Many serious diseases, like diabetes and HIV, are now much easier to treat than before.
  2. New treatments for conditions such as schizophrenia and allergies are being developed, showing promise for better ways to help people who suffer from these issues.
  3. Innovations like gene therapy and cancer vaccines are changing how we approach some of the most challenging health problems, potentially offering cures or significantly improved treatments.

In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice

Axial • 14 implied HN points • 23 Nov 24
🔬 Science Gene Therapy CRISPR Molecular Biology
  1. Researchers are using CRISPR technology to fix a genetic mutation that causes a type of blindness in mice. This mutation is in the rhodopsin gene, which is important for vision.
  2. Timing is really important for the treatment to be effective. Treating mice earlier resulted in better preservation of their eyesight compared to treating them later.
  3. Despite progress, there are challenges in translating this therapy for humans. The mutation is rare, making it tough to conduct clinical trials, and the therapy needs further development for success.

How to Engineer Therapeutic AAVs

LatchBio • 15 implied HN points • 14 Nov 24
🔬 Science Gene Therapy Bioinformatics Machine Learning
  1. Adeno-associated viruses (AAVs) are used for gene therapy because they can deliver therapeutic genes safely without causing disease in humans. They're like little delivery trucks that send important genetic information to specific parts of the body.
  2. Dyno Therapeutics created a new version of AAV called Dyno bCap1, which is much better at getting to the brain and avoiding the liver, showcasing how engineering can significantly improve these therapies.
  3. By using machine learning, scientists can design better AAVs by predicting how changes in their structure affect their ability to deliver genes. This makes the process smarter and helps create more effective treatments.
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Setting the Scene for Genetic Breakthroughs

Superfluid • 13 implied HN points • 15 Mar 23
🔬 Science Genetics Gene Therapy CRISPR Biotechnology
  1. Gene therapy has the potential to treat and cure genetic diseases, but it is a complex process requiring expertise in biology and manufacturing.
  2. The gene therapy value chain involves steps like target identification, payload design, delivery vehicle design, translation development, manufacturing, and clinical trials.
  3. Companies are working on breakthroughs in gene therapy technology, such as improving AAV vectors and refining CRISPR-Cas9 systems, but face challenges in experimental processes and data availability.