A reproducible and physiologically relevant human iPSC-derived platform . . . Cattelan and colleagues developed a neurocardiac co-culture using hiPSC-derived cells, addressing the lack of human cardiac innervation models The system replicates key neuro-cardiac mechanisms, showing hiPSC-sympathetic neurons' ability to control and modulate hiPSC-cardiomyocytes This innovative model offers a valuable platform for advancing disease modelling and testing new
Issue: Molecular Therapy - Cell Press The Food and Drug Administration (FDA) recently proposed a dedicated regulatory pathway for “personalized” therapies 1 targeting genetic variation found in small numbers of individuals This pathway is important, timely, and urgently needed A number of such interventions have been investigated—e g , milasen, a customized antisense oligonucleotide (ASO) drug designed for a child with
Dyslipidaemia-associated natural IgM improves oncolytic virus TILT-123 . . . Hemminki and colleagues examined samples from cancer patients treated with an oncolytic adenovirus and found that antibodies associated with elevated lipid levels bind both virus and modified lipids This interaction forms a virus–antibody complex that enhances viral uptake, transduction and tumour cell killing
AAV vector development, back to the future: Molecular Therapy Evolution of AAV libraries, from in vitro to rodents to large animals to ex vivo organs, still remains an unknown for success in humans Libraries in humans for humans is the next evolutionary step for AAV vector development