Ultrasound-activated Particles As Crispr/cas9 Delivery System For Androgenic Alopecia Therapy

Joxy

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Compared to a plasmid, viral, and other delivery systems, direct Cas9/sgRNA protein delivery has several advantages such as low off-targeting effects and non-integration, but it still has limitations due to low transfer efficiency. As such, the CRISPR/Cas9 system is being developed in combination with nano-carrier technology to enhance delivery efficiency and biocompatibility. We designed a microbubble-nanoliposomal particle as a Cas9/sgRNA riboprotein complex carrier, which effectively facilitates local delivery to a specific site when agitated by ultrasound activation. In practice, we successfully transferred the protein constructs into dermal papilla cells in the hair follicle of androgenic alopecia animals by microbubble cavitation induced sonoporation of our particle. The delivered Cas9/sgRNA recognized and edited specifically the target gene with high efficiency in vitro and in vivo, thus recovering hair growth. We demonstrated the topical application of ultrasound-activated nanoparticles for androgenic alopecia therapy through the suppression of SRD5A2 protein production by CRISPR-based genomic editing.

https://www.sciencedirect.com/science/article/pii/S0142961219308543?via=ihub
https://www.ncbi.nlm.nih.gov/pubmed/31901692

email: ghim@ajou.ac.kr
email: tjyoon@moogene.com

Maybe somebody can contact them and ask specific questions.

I hope so some very good news in future from these guys.
 

stachu

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Trichosan

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Compared to a plasmid, viral, and other delivery systems, direct Cas9/sgRNA protein delivery has several advantages such as low off-targeting effects and non-integration, but it still has limitations due to low transfer efficiency. As such, the CRISPR/Cas9 system is being developed in combination with nano-carrier technology to enhance delivery efficiency and biocompatibility. We designed a microbubble-nanoliposomal particle as a Cas9/sgRNA riboprotein complex carrier, which effectively facilitates local delivery to a specific site when agitated by ultrasound activation. In practice, we successfully transferred the protein constructs into dermal papilla cells in the hair follicle of androgenic alopecia animals by microbubble cavitation induced sonoporation of our particle. The delivered Cas9/sgRNA recognized and edited specifically the target gene with high efficiency in vitro and in vivo, thus recovering hair growth. We demonstrated the topical application of ultrasound-activated nanoparticles for androgenic alopecia therapy through the suppression of SRD5A2 protein production by CRISPR-based genomic editing.

https://www.sciencedirect.com/science/article/pii/S0142961219308543?via=ihub
https://www.ncbi.nlm.nih.gov/pubmed/31901692

email: ghim@ajou.ac.kr
email: tjyoon@moogene.com

Maybe somebody can contact them and ask specific questions.

I hope so some very good news in future from these guys.

Well, this is what needs to be known: What the hell were the protein constructs?!! They edited the gene to produce hair in vivo and in vitro and they're bragging about how they got it into the follice. WTF! This is like saying we found out why Brad Pitt doesn't lose his hair and grew some in a petri dish and on another blokes *** and look here's the shiny chrome syringe we used. Where's the full article????
 

Throwaway94

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The delivery system is the part we should be curious about, the fact that their gene edit is only 70% on-target is scary and it'll be (many many many) years before they can develop a safe and effective hair treatment out of it.

But should this become a reality wouldn't it be better to target the androgen receptor gene rather than the 5-ar2 gene? As this sort of local targeting would be of great benefit to men suffering prostate cancer it seems more likely to gain traction.
 
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