A small-molecule approach to restore female sterility phenotype targeted by a homing suppression gene drive in the fruit pest Drosophila suzukiidoi:10.1371/journal.pgen.1011226DROSOPHILA suzukiiGENE silencingPHENOTYPESGENETIC engineeringDROSOPHILA melanogaster...
A small molecule restores function to TRPML1 mutant isoforms responsible for mucolipidosis type IV Mutations in thegene are causative for MLIV. We used lead optimization strategies to identify—and MLIV patient fibroblasts to test—small-molecule activators for their potential to restore TRPML1 ...
Generian is building a first in class drug discovery pipeline of small molecule drugs to address the shortcomings of biologics
On today’s episode of the pharmaphorum podcast, Jason Park, CEO of Empress Therapeutics, discusses his company's novel approach to small molecule drug discovery.
Premature termination codons (PTCs) prevent translation of a full-length protein and trigger nonsense-mediated mRNA decay (NMD). Nonsense suppression (also termed readthrough) therapy restores protein function by selectively suppressing translation termination at PTCs. Poor efficacy of current readthrough ...
Using this assay, approximately 45,000 wells from various small molecule diversity and targeted libraries were tested for the ability to increase PINK-1/PINK1::GFP-signal. Primary hits were counter-screened in wild-type worms (which do not express GFP) to eliminate the possibility that the compo...
The PARP family consists of 17 subtypes, which are divided into DNA-dependent, Tankyrases, CCCH-Parps, Macro-parps and other-parps according to their function and structure. PARP inhibitors, which are small molecule inhibitors that hinder DNA repair, are thought to govern anticancer effects ...
To date, several synthetic-peptide-based inhibitors have been reported to be able to interfere with PB1–PB2 interactions and disrupt normal function of the polymerase91, 92. However, nonpeptide small-molecule based inhibitors remain to be discovered. One particular challenge is to design small-...
bleaching13,19,51. Pharmacological chaperones can mitigate the effects of misfolded proteins and restore function. An example is the recent discovery of a novel small-molecule stabilizer of clarin-1 that can rescue hearing in a mouse model carrying the clarin-1 mutation N48K commonly found in ...
This study demonstrates the viability of gene therapy for the haematopoietic system in FA patients, however the elevated cancer risk for the rest of the body3 would presumably remain high. Complementary approaches to gene therapy are also being investigated. There are clinical trials with metformin ...