Splicing is a process necessary for the correct synthesis of proteins and an essential step in gene expression. An impaired minor splicing may lead to aberrant pre-mRNA transcripts and exon skipping, leading to premature stop codons and truncated synthesized proteins, resulting in severe consequences.
In a recent study published in Nature Genetics, a team of scientists used CRISPR-Cas9 gene editing to systematically analyze genetic weaknesses in uveal melanoma cells and comprehensively map monogenic and digenic dependencies.
Rhabdomyosarcoma is the most frequent cancer affecting soft tissues in children, and existing therapies often fail to stop relapse or prolong survival. Researchers in Germany and the U.K. have developed a potential new therapy based on natural killer cells expressing a chimeric antigen receptor against epidermal growth factor receptor (EGFR-CAR).
A recent Merck Sharp & Dohme LLC patent details new substituted isoquinoline derivatives acting as tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and/or PTPN2 (TCPTP) inhibitors potentially useful for the treatment of cancer.
Work at Childrens Medical Center Corp. has led to the identification of new gasdermin-D (GSDMD) activators reported to be useful for the treatment of cancer.
Discoidin domain receptor tyrosine kinase 1 (DDR1) contributes to tumor progression by promoting the alignment and densification of collagen fibers within the extracellular matrix (ECM), thereby facilitating the development of an immune-excluded tumor microenvironment (TME).
A Black Diamond Therapeutics Inc. patent describes new alkyne-substituted quinazoline derivatives acting as EGFR (HER1; erbB1) inhibitors particularly, EGFR mutant inhibitors, potentially useful for the treatment of cancer.
Dysfunction of the tumor suppressor p53, commonly resulting from MDM2 overexpression or gene mutations, plays a key role in breast cancer progression. While the bioactive compound piperine has been shown to enhance p53 activity, its clinical utility is limited by poor bioavailability, potential toxicity and the risk of adverse drug interactions.