Scientist, cyclist, runner, and movies/books consumer. Views are my own.
San Francisco, CA
Joined August 2014
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A recent Perspective in Nature Medicine highlights how the rapid expansion of the antibody–drug conjugates (ADC) design space has generated more promising development candidates than can realistically be evaluated in patients.
lnkd.in/g9AJVXKQ
Circular RNA with embedded aptmers. Went strait to human testing after positive results on rodent models. Chinese biotech!
biorxiv.org/content/10.1101/…
#circRNA #aptamers #ChineseBiotech
Base Editors make double-stranded brakes and chromosome rearangements
#ESGCT2025 #BaseEditors #OffTagets
Clinical limitations of AAV microdystrophin gene therapy
Jeffrey S. Chamberlain from
@uwsomwwami
#ESGCT2025 #DMD #GeneTgerapy
Efficient EGFP reporter transgene delivery to NHP T cells by LNP. Next step is delivering the CAR in vivo
#ESGCT2025 #InVivoCART
Cecilia Cotta-Ramusino @TesseraTx
Liver directed Klotho overexpression fails to protect from ALS in mice
Sergi Verdes, Universitat Autonoma de Barcelona
#ALS #GeneTherapy #Klotho
ZFP genome editors for suppressing prion gene expression. Surprisingly similar to @SangamoTx
@DevermanLab
#EpigenomeEdirors #prion ##ESGCT2025
StichR technology for expressing minidystrophin in mouse skeletal muscle. Douglas Anderson from @UR_Med
#ESGCT2025 #DMD ##GeneTherapy
Official press release for @SonotheraI41798 presentation @ #ESGCT2025.
Results show high levels of durable human dystrophin protein expression exceeding
50% of endogenous levels have been achieved in NHP.
Poster Session 2, Oct 9th. Poster P0266.
#Dmd #GeneTherapy #nonviral
The ability to deliver targeted, full-length human dystrophin to skeletal, heart and diaphragm muscles in a durable, safe, redosable and noninvasive manner shows how UMD can overcome all of the challenges of current gene therapy delivery options. Learn more: businesswire.com/news/home/2… #ESGCT2025 #newdata #geneticmedicine #ultrasound #microbubble #nonviral #duchennemusculardystrophy
Heading to #ESGCT2025 to share the @SonotheraI41798 team’s latest advances in non-viral delivery of gene therapy vectors expressing full-length dystrophin gene.
#DMD #GeneTherapy #Nonviral
Ivan Krivega retweeted
Interesting observations on Enhancer-Gene (E-G) interactions in single-cells from scSPRITE
1. Nanog: if long-range E-G interactions present, short range E-G interactions less likely
2. Tbx3: long-range E-G interactions anticorrelate with ~TAD structures
biorxiv.org/content/10.1101/…
IdeS treatment before #AAV vector infusion was safe and resulted in enhanced liver transduction, even in the setting of vector readministration in NHP.
nature.com/articles/s41591-0…
Fusion of the DNA-binding domain of RAD51 to CRISPR base editors enhances efficiency and targeting range.
#CRISPR #BaseEditing
nature.com/articles/s41556-0…
nature.com/articles/s41556-0…
New iteration of p53/#CRISPR story.
Introduction of Cas9 can lead to the emergence and expansion of p53-inactivating mutations.
Still not clear if thisis true for anothet #GeneEditing tools
#Cancer
nature.com/articles/s41588-0…
New #CRISPR increase the rate of HDR repair by 2-2.5 fold and decrease cellular toxicity by 2-4 fold.
biorxiv.org/content/10.1101/…
#GeneEditing
