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Images showing the green fluorescence signals in different body parts of the live-birth chimeric monkey.
Drug Design, Drug Delivery & Technologies

1st chimeric monkey born with large embryonic stem cell contribution

Nov. 9, 2023
By Anette Breindl
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.
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3D illustration of heart cross section
Genetic/Congenital

AAV-RBM20 gene therapy corrects splicing in cardiomyopathy model

Oct. 30, 2023
Approximately 2% to 6% of familiar dilated cardiomyopathy (DCM) cases are caused by inherited mutations in the RBM20 gene, which encodes the RNA binding motif 20 (RBM20), a splicing factor that regulates the splicing of several targets involved in the regulation of sarcomeric structure and calcium handling in the myocardium.
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Neurology/Psychiatric

A new exon-skipping strategy for treating Duchenne muscular dystrophy

Oct. 27, 2023
Duchenne muscular dystrophy (DMD) is a disorder characterized by progressive degeneration of muscles, resulting in muscle loss, mobility limitations and lately, mortal risk. DMD is caused by mutations in the dystrophin gene (DMD) and about 80% of these are suitable for exon skipping, obtaining a functional dystrophin protein.
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Neurology/Psychiatric

Gene editing approaches for the treatment of Huntington’s disease and ALS by Huidagene Therapeutics

Oct. 26, 2023
Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
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Vial and syringe with DNA
Drug Design, Drug Delivery & Technologies

In HIV, gene therapy could be alternative path to functional cure

Oct. 26, 2023
By Anette Breindl
At the 30th Annual Congress of the European Society for Gene and Cell Therapy in Brussels this week, researchers presented both preclinical and clinical strategies for applying gene therapy to a functional HIV cure. At a Wednesday session on Infectious Diseases & Vaccines, Alessio Nahmad, of Tabby Therapeutics Ltd., described using B cells edited to express broadly neutralizing antibodies (bnAbs) 3BNC117 to deliver high titers of antibodies in mice.
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