Gene therapy has long held promise to correct a variety of human diseases and defects.
Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the …

Vaccines represent the single most cost-efficient and equitable way to combat and eradicate
infectious diseases. While traditional licensed vaccines consist of either …

Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes …

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that
is caused by a deficiency in ALD protein, an adenosine triphosphate–binding cassette …

To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional
gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Human diseases, particularly infectious diseases and cancers, pose unprecedented
challenges to public health security and the global economy. The development and …

Gene transfer into hematopoietic stem cells has been used successfully for correcting
lymphoid but not myeloid immunodeficiencies. Here we report on two adults who received …