[EN] POLYNUCLEOTIDES ENCODING JAGGED1 FOR THE TREATMENT OF ALAGILLE SYNDROME<br/>[FR] POLYNUCLÉOTIDES CODANT POUR JAGGED1 POUR LE TRAITEMENT DU SYNDROME D'ALAGILLE
申请人:MODERNATX INC
公开号:WO2017201342A1
公开(公告)日:2017-11-23
The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof, functional fragments thereof, and fusion proteins comprising JAG1, mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.
[EN] POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS<br/>[FR] POLYNUCLÉOTIDES CODANT POUR UN RÉGULATEUR DE CONDUCTANCE TRANSMEMBRANAIRE DE FIBROSE KYSTIQUE POUR LE TRAITEMENT DE LA FIBROSE KYSTIQUE
申请人:MODERNATX INC
公开号:WO2017201347A1
公开(公告)日:2017-11-23
The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.
A highly efficient method for the hydroaminomethylation of long-chain alkenes under aqueous, biphasic conditions
作者:Arno Behr、Marc Becker、Sebastian Reyer
DOI:10.1016/j.tetlet.2010.02.145
日期:2010.5
secondary and primary amines with different inorganic and organic acids in hydroaminomethylation enables the quantitative conversion of 1-octene with high selectivity for saturatedamines. We propose that a cationic rhodium species is formed under the acidic conditions which catalyses the hydrogenation of the enamine or imine formed subsequently. Thus the use of acids and amine salts enables the hydroaminomethylation