First Positive Data of In Vivo CRISPR-Based Genome Editing in Humans Reported
June 28, 2021
A team of researchers based in the U.S., the U.K., and New Zealand from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners showed in a study that the companies’ lead
in vivo genome editing candidate NTLA-2001 generated a dose-dependent sustained reduction of protein linked to transthyretin (ATTR) amyloidosis following a single dose in six patients living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
The study is the first ever to support the safety and efficacy of
in vivo CRISPR genome editing in humans.
The New England Journal of Medicine and in a presentation that day at the 2021 Peripheral Nerve Society (PNS) Annual Meeting the researchers reported that a single 0.3 mg/kg dose of NTLA-2001 led to an 87% mean reduction in serum transthyretin (TTR) protein concentration in three of the ATTRv-PN patients by day 28, with individual re
OHCHR | Second UN high Level Conference on Counter terrorism
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New Era of Medicine : Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans
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In collaboration with the Permanent Mission of the Federal Republic of Germany to the United Nations and Global Project Against Hate and Extremism, the World Jewish Congress is organizing a virtual event entitled “Trends in Financing Far-Right Violent Extremism” on 28 June.
The event will explore the confluence of the coronavirus pandemic and the financing of
far-right violent extremism, in particular discussing the influence
of new technologies and online capabilities such as the improper
use of cryptocurrency, the selling of hate merchandise and the use of social
media for financing.
The
event will take place in the framework of the Second United