A broad spectrum of human neurological diseases linked to variations in one gene, DHX9

A broad spectrum of human neurological diseases linked to variations in one gene, DHX9

An international team of researchers from the GREGoR Research Center at Baylor College of Medicine, the Chinese University of Hong Kong, the German Mouse Clinic and collaborating institutions have provided a genetic diagnosis to a group of 20 patients with hitherto undiagnosed neurological diseases. The team analyzed the patients’ genes and conducted family studies to … Read more

Cure Rare Disease identifies the toxicity responsible for DMD gene therapy death

Cure Rare Disease identifies the toxicity responsible for DMD gene therapy death

This content originally appeared on our sister site,CGTLIVE. According to a recent non-peer-reviewed research preprint, the Cure Rare Disease (CRD) investigations clarified that a gene therapy induced innate immune signaling with capillary-leakage-limiting toxicity, leading to patient death in a single clinical study (NCT05514249).1 The Duchenne muscular dystrophy (DMD) patient was treated with a CRISPR-based adeno-associated … Read more

Malaria unveiled: decoding the control of parasite gene expression

Researchers at Tokyo Tech’s World Research Hub Initiative conducted a study to investigate the regulation of gene expression in the apicoplast of the malaria parasite Plasmodium falciparum. Researchers at Tokyo Tech’s World Research Hub Initiative conducted a study to investigate the regulation of gene expression in the malaria parasite’s apicoplast Plasmodium falciparum. The results have … Read more

Could CRISPR gene therapy help treat Alzheimer’s disease?

A close up of an older adult woman holding a vase with a lily inside

Share on PinterestGene-editing technology may help scientists create new treatments for Alzheimer’s. Olga Sibirskaja/Stocksy Two innovative CRISPR-based therapeutic approaches for Alzheimer’s disease were unveiled at the Alzheimers Association International Conference (AAIC) 2023 in Amsterdam. One method targets the APOE-e4 gene, a major genetic risk factor for Alzheimer’s, with the aim of mitigating its effects. The … Read more

The family comes into contact with the researchers behind Canavan gene therapy

Terence R. Fleet;  Guangpinggao;  Lori, Lee and Noa Greenwood;  and Dominic Gesler

Terence R. Fleet; Guangpinggao; Lori, Lee and Noa Greenwood; and Dominic Gessler during a visit to UMass Chan earlier this year. A Boston boy who was one of the first to receive an experimental gene therapy for Canavan disease developed at UMass Chan Medical School expressed a very special “thank you” to the research team … Read more