Treatments for rare diseases are hard to create and expensive to deliver, but there is new hope for editing the software of the genome.

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Correcting Genetic Spelling Errors With Next-Generation Crispr

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Bay Area biotech Profluent has taken another step in using AI to design new CRISPR gene editing systems, CEO Ali Madani exclusively told Endpoints News.
The startup posted a preprint Monday ...

Endpoints News

Profluent debuts AI model for CRISPR design, hoping to expand gene editing's potential

(Wired) – Treatments for rare diseases are hard to create and expensive to deliver, but there is new hope for editing the software of the genome. For progeria and thousands of other genetic diseases, there are two reasons why this same approach won’t work. First, the desired edit for most misspellings will not usually be achieved by a disabling cut in the gene. Instead, a correction is needed.  In the case of progeria, the disease-causing T need…

Bioethics.com

With the help of the CRISPR gene scissors, a team led by Simone Spuler from the Experimental and Clinical Research Center in Berlin wants to develop a targeted therapy for muscular dystrophy. They are now presenting their preclinical results in "Nature Communications". They pave the way for the first studies on humans.
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Mithilfe der Genschere CRISPR will ein Team um Simone Spuler vom Experimental and Clinical Research Center in Berlin eine gezielte Therapie für Muskeldystrophie entwickeln. Ihre präklinischen Ergebnisse stellen sie nun in „Nature Communications“ vor. Sie ebnen den Weg für erste Studien am Menschen.
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Mit CRISPR gegen Muskelschwund

bionity.com

Using CRISPR to Combat Muscle Atrophy

Sam Berns is my friend. With his sage wisdom, he inspired me and many others on how to make the most of life. Suffering from a rare disease called progeria, he aged rapidly, and died of heart failure when he was only 17 years old, his brave life was cut too short. letter gone awry, T which should be C in a critical gene called lamin A. The same spelling is found in almost all 200 individuals around the world with progeria. The opportunity to ove…

Correcting Genetic Spelling Errors With Next-Generation Crispr

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