Gene Therapy

Scientists have discovered a set of surprisingly simple rules that determine the precision of CRISPR/Cas9 genome editing in human cellsCredit: Nigel Hawtin for the Francis Crick Institute Scientists at the Francis Crick Institute have…

Personalised medicine expert is the only Singapore-based academic inventor to receive this esteemed professional accolade this yearCredit: National University of Singapore Professor Dean Ho, Director of the Singapore Institute for…

For the first time ever, biomedical researchers from Aarhus University, Denmark, have unveiled cellular defects leading to the rare disease hereditary angioedema (HAE). This new understanding is an important step towards gene therapy for…

First patient treated in the European clinical trial sponsored by Genethon Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon,…

The article by Dr. Yunfeng Lin et al. is published in Current Stem Cell Research & Therapy, Volume 13, Issue 7, 2018 Chondral defect is a kind of defect which refers to focal area damage to the articular cartilage. This defect also…

The article by Dr. Gary Hardiman et al. is published in Current Genomics, Volume 19, Issue 7, 2018Credit: Dr. Gary Hardiman et al., Bentham Science Publishers Cellular pathways represent the intricate metabolic connections between the…

RCSI researchers play key role in international collaborationCredit: RCSI Monday, 10 December 2018: The largest study of its kind, led by international researchers including scientists at RCSI (Royal College of Surgeons in Ireland), has…

Team of RI-MUHC researchers discover new disease and its genetic cause in Quebec patient as 'Bubble Boy' symptoms discovered in adults for first timeCredit: RI-MUHC A team from the Research Institute of the McGill University Health Centre…

Study findings can immediately be applied to human patients with the diseaseCredit: Patricia M. Zerfas, NIH Office of Research Services. Researchers have discovered that a hormone, fibroblast growth factor 21 (FGF21), is extremely…

CHOP celebrates landmark medical advance: the first gene therapy for a genetic disease approved in both the US and Europe Children's Hospital of Philadelphia (CHOP) celebrates a pivotal moment in medicine: approval by the European…