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 …

Therapeutic strategies are being clinically tested either to eradicate latent HIV reservoirs or
to achieve virologic control in the absence of antiretroviral therapy. Attaining this goal will …

Chimaeric antigen receptor (CAR) T cells can generate durable clinical responses in B-cell
haematologic malignancies. The manufacturing of these T cells typically involves their …

Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy
and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine …

Affinity-optimized T cell receptors can enhance the potency of adoptive T cell therapy.
Afamitresgene autoleucel (afami-cel) is a human leukocyte antigen-restricted autologous T …

A stable latent reservoir for HIV-1 in resting CD4+ T cells is the principal barrier to a cure,–.
Curative strategies that target the reservoir are being tested, and require accurate, scalable …

We report a patient relapsing 9 months after CD19-targeted CAR T cell (CTL019) infusion
with CD19–leukemia that aberrantly expressed the anti-CD19 CAR. The CAR gene was …

Abstract Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-
matched donor may benefit from gene therapy through the provision of gene-corrected …

Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells,. We
report the initial results of nine severely affected X-linked CGD (X-CGD) patients who …

Clonal expansion of infected CD4+ T cells is a major mechanism of HIV-1 persistence and a
barrier to achieving a cure. Potential causes are homeostatic proliferation, effects of HIV-1 …