Gene therapy (GT) has tremendous potential for the treatment of neurological disorders to
transform patient care. The successful application of virus-mediated GT to treat spinal …

Following the birth in 2018 of two babies from embryos altered using CRISPR-Cas9, human
germline gene editing (GGE) moved from abstract concern to reality. He Jiankui, the scientist …

Background Forward-looking, democratically oriented governance is needed to ensure that
human genome editing serves rather than undercuts public values. Scientific, policy, and …

Rare genetic diseases are a group of life-threatening disorders affecting significant
populations worldwide and posing substantial challenges to healthcare systems globally …

Background and Objective: New and more efficient methods of gene editing have intensified
the ethical and legal issues associated with editing germlines. Yet no research has …

Virus-specific T cells have proven highly effective for the treatment of severe and drug-
refractory infections after hematopoietic stem cell transplant (HSCT). However, the efficacy of …

The CRISPR/Cas system could provide an efficient and reliable means of editing the human
genome and has the potential to revolutionize modern medicine; however, rapid …

The advent of CRISPR-Cas9 in 2012 started revolutionizing the field of genetics by
broadening the access to a method for precise modification of the human genome. It also …

Public participation, transparency and accountability are three of the pillars of good
governance. These pillars become particularly important for innovative, personalised health …

STAT3-hyper IgE syndrome (STAT3-HIES) is a primary immunodeficiency presenting with
destructive lung disease along with other symptoms. CRISPR-Cas9-mediated adenine base …