Gene therapy strategy found effective in mouse model of hereditary disease TSC

Journal Reference: Pike-See Cheah, Shilpa Prabhakar, David Yellen, Roberta L. Beauchamp, Xuan Zhang, Shingo Kasamatsu, Roderick T. Bronson, Elizabeth A. Thiele, David J. Kwiatkowski, Anat Stemmer-Rachamimov, Bence György, King-Hwa Ling, Masao Kaneki, Bakhos A. Tannous, Vijaya Ramesh, Casey A. Maguire, Xandra O. Breakefield. Gene therapy for tuberous sclerosis complex type 2 in a mouse model … Read more

A genetic patch to prevent hereditary deafness

Journal Reference: Erik de Vrieze, Jorge Cañas Martín, Jolien Peijnenborg, Aniek Martens, Jaap Oostrik, Simone van den Heuvel, Kornelia Neveling, Ronald Pennings, Hannie Kremer, Erwin van Wijk. Antisense oligonucleotide-based degradation of c.151C>T mutant COCH transcripts associated with dominantly inherited hearing impairment DFNA9.. Molecular Therapy – Nucleic Acids, 2021; DOI: 10.1016/j.omtn.2021.02.033 Hereditary deafness can manifest itself … Read more