On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …

Intracellular delivery of materials has become a critical component of genome-editing
approaches, ex vivo cell-based therapies, and a diversity of fundamental research …

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an
excellent safety record. They provide evidence for the long-sought promise of gene therapy …

Background In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function
of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and …

The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by
mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene …

Genome editing via homologous recombination (HR)(gene targeting) in human
hematopoietic stem cells (HSCs) has the power to reveal gene–function relationships and …

Abstract ß-thalassemia is caused by ß-globin gene mutations resulting in reduced (β+) or
absent (β0) hemoglobin production. Patient life expectancy has recently increased, but the …

Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …