Acute myeloid leukemia (AML) harboring KMT2A rearrangements (KMT2A-r) represents a highly aggressive disease subtype, characterized by poor therapeutic response and a high risk of relapse.
DNA damage repair enzymes are interesting targets in cancer, since genomic instability and DNA repair defects are important cancer cell hallmarks. Poly (ADP-ribose) glycohydrolase (PARG) is the dominant eliminator of PARylation in the cell, the activity of which prevents excessive accumulation of PARylation, and promotes the dissociation of repair proteins, as well as ensuring the smooth completion of DNA repair process.
Werner syndrome ATP-dependent helicase (WRN) is an enzyme involved in DNA replication and repair and has been identified as a synthetic lethality target in tumors with high microsatellite instability (MSI-H).
Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
CCR8 is highly expressed on immunosuppressive regulatory T cells (Tregs) in various solid tumors, making it a potential target to enhance antitumor immunity and the efficacy of cancer therapies, including checkpoint inhibitors. However, the impact of CCR8 expression on the Treg phenotype and its role in cancer progression remain unclear.
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
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