There have been many ups and downs since the introduction of gene therapy as a
therapeutic modality for diseases. However, the journey of gene therapy has reached a …

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 …

Ionizable lipid nanoparticles (LNPs) are the most clinically advanced nonviral platform for
mRNA delivery. While they have been explored for applications including vaccines and …

Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …

Due to their efficient recognition and lysis of malignant cells, natural killer (NK) cells are
considered as specialized immune cells that can be genetically modified to obtain capable …

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 …

Chimeric antigen receptor (CAR) T‐cell therapy represents a major advancement in
personalized cancer treatment. In this strategy, a patient's own T cells are genetically …

Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is
now commonplace in academic laboratories and industry for both research and clinical …

Junctional epidermolysis bullosa (JEB) is a severe and often lethal genetic disease caused
by mutations in genes encoding the basement membrane component laminin-332 …

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 …