Forkhead box protein L2 (FOXL2) is a protein involved in ovary development and maintenance, among other functions. Mutations in its gene are involved in ovarian failure and granulosa cell tumors, being the latter a rare cancer type caused in most cases by the C134W mutation (c.402C>G) in the FOXL2 gene. By using CRISPR/Cas9 technology, researchers from Spain have aimed to remove this mutation in the FOLX2 gene from granulosa tumor cells.

Researchers from Universidade Estadual da Paraíba, Universidade Federal da Paraiba and affiliated organizations presented the design, synthesis and bio-evaluation of new small-molecule candidates for the treatment of leishmaniasis, a neglected tropical disease that affects approximately 12 million people in four continents.

Venetoclax, a selective Bcl-2 inhibitor, has proven effective in chronic lymphocytic leukemia (CLL), but genetic mutations or adaptive changes over time can result in resistance to treatment and disease relapse. Researchers from Newave Pharmaceutical Inc. and collaborators described the preclinical efficacy of LP-118, a Bcl-2 inhibitor, in venetoclax-resistant models of CLL.

The absent, small or homeotic-like 1 (ASH1L) protein regulates the expression of HOXA genes, which are critical for the development of MLL1 translocations frequently found in leukemia patients. Knockdown of ASH1L leads to growth arrest and apoptosis of leukemia cells, thereby inhibiting leukemia progression suggesting that ASH1L can be considered as a therapeutic target in this setting.

Chinese researchers and their collaborators have published data on the bifunctional antibody JS-201, which exerts dual targeting of PD-1 and TGF-β signaling, for the potential treatment of cancer and its protective role against radiation-induced lung injury.

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud University developed a new antisense oligonucleotide (AON) therapy, designed to rescue the splicing defect caused by this variant.

Scientists at the University of Pittsburgh have tested a broadly neutralizing antibody that binds the stem of a protein against H5N1 avian influenza, which prevented severe disease in nonhuman primates and was effective for at least 8 weeks. The scientists remark on the potential of the design developed by Astrazeneca plc against a possible outbreak of the influenza A virus.