ShapeTX uses a guide RNA that can recruit the cell’s own adenosine deaminase acting on RNA (ADAR) enzymes to edit a precise location on a target RNA. ADARs can correct guanine-to-adenine (G-to-A ...
ProQR Therapeutics is advancing its Axiomer ADAR-mediated RNA editing pipeline, aiming to file a Clinical Trial Application for its lead program AX-0810, targeting NTCP for cholestatic diseases ...
This form of RNA editing is conducted by an enzyme called ADAR (Adenosine Deaminases Acting on RNA), altering one of the molecules within double-stranded RNA, converting it from adenosine (A ...
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for ...
Therapeutic RNA editing platforms based on this mechanism fall into one of two categories: either they use engineered enzymes, which generally consist of the editing part of the ADAR enzyme attached ...
Reported in a 2019 Nature Biotechnology paper, this technology, called ‘leveraging endogenous ADAR for programmable editing of RNA’, or LEAPER, can be a safe gene-editing tool, according to Wei.
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases ...
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company ...
As we continue to advance our Axiomer ADAR-mediated RNA editing platform and pipeline, ProQR is entering a pivotal phase of growth and ...
KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the ...
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