CRISPR gene-editing breakthrough opens door to treating broad array of diseases
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Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#2Off topic: are there any central repositories tracking gene therapy progress for various genetic conditions, with a similar UX to Moderna’s mRNA pipeline tracker?
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#3I must be out of the loop. Last I heard trials with CRISPR were making not only the desired genetic changes but also dozens of others at random points in the genome. Did they solve that problem?
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#4The period missing from the title makes this very hard to parse what is being said.
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#5I must be out of the loop. Last I heard trials with CRISPR were making not only the desired genetic changes but also dozens of others at random points in the genome. Did they solve that problem?
Humans improve technology all the time, CRISPR gene editing would be no exception.
Straight from Wikipedia: https://en.wikipedia.org/wiki/LEAPER_gene_editing
I bet a dip into the specialized papers will show a multitude of these techniques.
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#6Original paper: https://www.nejm.org/doi/full/10.1056/NEJMoa2107454?query=fe...
This is the first published IV-administration of a gene-editing CRISPR payload. Interesting convergence with the nanoparticles used in mRNA vaccines. Sickle-cell and immune reprogramming edits have been made on cells removed from the body. Josiah Zayner infamously injected himself with CRISPR at a conference years back, but (1) unpublished, AFAIK and (2) he had been drinking which likely attenuated the payload [and (3) he wanted bigger muscles, not to treat a disease]
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#7Wow, it worked...This uses LNPs similar to the recent Moderna and BioNTech/Pfizer vaccines, and N1m-pseudouridine mRNA, just like the mRNA vaccines. This is incredible.
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#8Reminds me of this guy that cured himself of lactose intolerance. https://www.popularmechanics.com/science/health/a17804128/sc...
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#9What are the limits of gene editing?
Re: CRISPR gene-editing breakthrough opens door to treating broad array of diseases
#10Off topic: are there any central repositories tracking gene therapy progress for various genetic conditions, with a similar UX to Moderna’s mRNA pipeline tracker? https://www.modernatx.com/pipeline
All FDA trials are listed here: https://clinicaltrials.gov/, I believe you can search by gene. It's not as nice as the moderna pipeline though.