• OTS
  • April 23, 2021
SHARE
Post Views: 1,051

Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.

Shao Y, Sztainberg Y, Wang Q, Bajikar SS, Trostle AJ, Wan YW, Jafar-Nejad P, Rigo F, Liu Z, Tang J, Zoghbi HY.

Sci Transl Med. 2021 Mar 3;13(583):eaaz7785.

Pubmed

Related articles

Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome

Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome

April 23rd, 2021
α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

April 23rd, 2021
Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins

Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins

March 15th, 2021
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

March 15th, 2021
Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

March 15th, 2021
Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)

Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)

February 16th, 2021