期刊: MOLECULAR THERAPY, 2022; 30 (1)
Machado-Joseph disease (MJD) is a fatal neurodegenerative disorder clinically characterized by prominent ataxia. It is caused by an expansion of a CAG......
期刊: MOLECULAR THERAPY, 2022; 30 (1)
B cells have been engineered ex vivo to express an HIV-1 broadly neutralizing antibody (bNAb). B cell reprograming may be scientifically and therapeut......
期刊: MOLECULAR THERAPY, 2022; 30 (1)
Compact CRISPR-Cas9 systems that can be packaged into an adeno-associated virus (AAV) show promise for gene therapy. However, the requirement of proto......
期刊: MOLECULAR THERAPY, 2022; 30 (1)
Adrenoleukodystrophy (ALD) is caused by various pathogenic mutations in the X-linked ABCD1 gene, which lead to metabolically abnormal accumulations of......
期刊: MOLECULAR THERAPY, 2022; 30 (2)
Despite increasing interest in the reversal of age-related processes, there is a paucity of data regarding the effects of post-menopausal-associated e......
期刊: MOLECULAR THERAPY, 2022; 30 (3)
STAT3 signaling has been shown to regulate cellular function and cytokine production in the tumor microenvironment (TME). Within the head and neck squ......