The present invention provides compounds, compositions thereof, and methods of using the same.
本发明提供了化合物、其组合物以及使用这些化合物的方法。
[EN] METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE<br/>[FR] PROCÉDÉS ET COMPOSÉS POUR LE TRAITEMENT D'UNE MALADIE GÉNÉTIQUE
申请人:DESIGN THERAPEUTICS INC
公开号:WO2021158707A1
公开(公告)日:2021-08-12
The present disclosure relates to compounds and methods for modulating the expression of dmpk, and treating diseases and conditions in which dmpk plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CAG or CTG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CAG or CTG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.
[EN] REGULATING CHIMERIC ANTIGEN RECEPTORS<br/>[FR] RÉGULATION DE RÉCEPTEURS D'ANTIGÈNES CHIMÉRIQUES
申请人:DANA FARBER CANCER INST INC
公开号:WO2018148440A1
公开(公告)日:2018-08-16
This invention is in the area of compositions and methods for regulating chimeric antigen receptor immune effector cell, for example T-cell (CAR-T), therapy to modulate associated adverse inflammatory responses, for example, cytokine release syndrome and tumor lysis syndrome, using targeted protein degradation.
[EN] TUNABLE ENDOGENOUS PROTEIN DEGRADATION WITH HETEROBIFUNCTIONAL COMPOUNDS<br/>[FR] DÉGRADATION MODULABLE DE PROTÉINE ENDOGÈNE AVEC DES COMPOSÉS HÉTÉROBIFONCTIONNELS
申请人:DANA FARBER CANCER INST INC
公开号:WO2018148443A1
公开(公告)日:2018-08-16
The present invention provides a means to modulate gene expression in vivo in a manner that avoids problems associated with CRISPR endogenous protein knock-out or knock-in strategies and strategies that provide for correction, or alteration, of single nucleotides. The invention includes inserting into the genome a nucleotide encoding a heterobifunctional compound targeting protein (dTAG) in-frame with the nucleotide sequence of a gene encoding an endogenously expressed protein of interest which, upon expression, produces an endogenous protein-dTAG hybrid protein. This allows for targeted protein degradation of the dTAG and the fused endogenous protein using a heterobifunctional compound.
Vortex-Induced Alignment of a Water Soluble Supramolecular Nanofiber Composed of an Amphiphilic Dendrimer
作者:Taiki Yamamoto、Akihiko Tsuda
DOI:10.3390/molecules18067071
日期:——
We have synthesized a novel amphiphilic naphthalene imide bearing a cationic dendrimer wedge (NID). NID molecules in water self-assemble to form a two-dimensional ribbon, which further coils to give a linear supramolecular nanofiber. The sample solution showed linear dichroism (LD) upon stirring of the solution, where NID nanofibers dominantly align at the center of vortex by hydrodynamic interaction with the downward torsional flows.