 Home > Biochemistry News > Biotechnology News > In vivo gene editing achieves another breakthrough, first children's clinical results released

In vivo gene editing achieves another breakthrough, first children's clinical results released

 Last Update: 2021-11-16
 Source: Internet
 Author: User

Tags

entresto free trial

update strontium citrate clinical trial

Search more information of high quality chemicals, good prices and reliable suppliers, visit www.echemi.com

Today, LogicBio Therapeutics announced the preliminary results of a Phase 1/2 clinical trial of its in vivo editing therapy LB-001 for the treatment of children with methylmalonic acidemia (MMA)

The press release states that this is the first clinical trial that has shown successful gene editing in children

In vivo gene editing achieved a major breakthrough this year.

The design of LB-001 uses an AAV vector to precisely insert a transgene expressing methylmalonyl-CoA mutase (MMUT) into the albumin gene locus of liver cells

The AAV vector also carries a transgene expressing albumin-2A.

▲The mechanism of action of LB-001 (picture source: LogicBio Therapeutics official website)

Reference materials:

[1] LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children.

(The original text has been deleted)

This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.

Related Products

L-Ascorbate-2-Monophosphate Feed Grade

CAS No.: 109620-90-8

Feed Grade

Potassium Sorbate from reliable manufacturer

CAS No.: 590-00-1

Food Grade

Doxycycline Hydrochloride

CAS No.: 10592-13-9

Pharmaceutical Grade

Hot Tags

united states jobs(223)

jobs united states(236)

1 year treasury(277)

1 year subscription(296)

announcements today(81)

80 20 company(94)

united states logo(227)

single use technology(195)

healthy food name(42)

ups united states(221)

Hot Articles

The source of this page with content of products and services is from Internet, which doesn't represent ECHEMI's opinion. If you have any queries, please write to service@echemi.com. It will be replied within 5 days.

Moreover, if you find any instances of plagiarism from the page, please send email to service@echemi.com with relevant evidence.