期刊： GENE THERAPY, 2023; 30 (7-8)
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype five gene therapy under investigation for the treatment of hemophili......
期刊： GENE THERAPY, 2023; 30 (1-2)
Recombinant adeno-associated virus (AAV) vectors are the leading platform for gene delivery for a variety of clinical applications. Patients with pree......
Fibroblast growth factor 21 (FGF21) has been developed as a potential therapeutic agent for metabolic syndromes. Moreover, FGF21 is considered a pro-l......
期刊： GENE THERAPY, 2023; 30 (3-4)
Human adipose-derived mesenchymal stem cells (ASCs) transduced with a lentiviral vector system to express bone morphogenetic protein 2 (LV-BMP-2) have......
Adeno-associated virus (AAV) vector gene therapy is a promising approach to treat rare genetic diseases; however, an ongoing challenge is how to best ......
Gene therapy is a new class of medical treatment that alters part of a patient's genome through the replacement, deletion, or insertion of genetic mat......
期刊： GENE THERAPY, 2023; 30 (6)
Protein misfolding and aggregation are shared features of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), and protein quali......
期刊： GENE THERAPY, 2023; 30 (6)
Transcription of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is regulated by both ubiquitous and cell-type selective cis-regul......
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for in......
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects motor neurons, causing progressive muscle weakness and respirato......
期刊： GENE THERAPY, 2023; 30 (1-2)
Intracerebral hemorrhage (ICH) is a life-threatening condition with a high mortality rate. For survivors, quality of life is determined by primary and......
期刊： GENE THERAPY, 2023; 30 (7-8)
Immune responses to Cas proteins have been demonstrated recently and these may prove to be an impediment to their clinical use in gene editing. To mak......
期刊： GENE THERAPY, 2023; 30 (3-4)
Gene delivery via focused ultrasound (FUS) mediated blood-brain barrier (BBB) opening is a disruptive therapeutic modality. Unlocking its full potenti......
期刊： GENE THERAPY, 2023; 30 (1-2)
Gut bacteria-associated sepsis is a serious concern in patients with gastrointestinal acute radiation syndrome (GIARS). In our previous studies, all m......
