Viral vector platforms within the gene therapy landscape
Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …
transitions. It has seen great strides in combating human disease, has given hope to patients …
Evaluating the state of the science for adeno-associated virus integration: an integrated perspective
DE Sabatino, FD Bushman, RJ Chandler, RG Crystal… - Molecular Therapy, 2022 - cell.com
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 …
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
Hematologic cancer after gene therapy for cerebral adrenoleukodystrophy
CN Duncan, JR Bledsoe, B Grzywacz… - … England Journal of …, 2024 - Mass Medical Soc
Background Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous
CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti …
CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti …
Engineered T cells: the promise and challenges of cancer immunotherapy
The immune system evolved to distinguish non-self from self to protect the organism. As
cancer is derived from our own cells, immune responses to dysregulated cell growth present …
cancer is derived from our own cells, immune responses to dysregulated cell growth present …
Recent advances in miRNA delivery systems
I Dasgupta, A Chatterjee - Methods and protocols, 2021 - mdpi.com
MicroRNAs (miRNAs) represent a family of short non-coding regulatory RNA molecules that
are produced in a tissue and time-specific manner to orchestrate gene expression post …
are produced in a tissue and time-specific manner to orchestrate gene expression post …
Gene therapy returns to centre stage
L Naldini - Nature, 2015 - nature.com
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 …
excellent safety record. They provide evidence for the long-sought promise of gene therapy …
Enhancement of the viability of T cells electroporated with DNA via osmotic dampening of the DNA-sensing cGAS–STING pathway
J An, CP Zhang, HY Qiu, HX Zhang, QB Chen… - Nature Biomedical …, 2024 - nature.com
Viral delivery of DNA for the targeted reprogramming of human T cells can lead to random
genomic integration, and electroporation is inefficient and can be toxic. Here we show that …
genomic integration, and electroporation is inefficient and can be toxic. Here we show that …
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013 - science.org
Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
A Aiuti, L Biasco, S Scaramuzza, F Ferrua, MP Cicalese… - Science, 2013 - science.org
Introduction Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency characterized
by eczema, thrombocytopenia, infections, and a high risk of developing autoimmunity and …
by eczema, thrombocytopenia, infections, and a high risk of developing autoimmunity and …