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ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease

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......

JIF:8.335

Reprogramming of the heavy-chain CDR3 regions of a human antibody repertoire

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......

JIF:8.335

Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9

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......

JIF:8.335

In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment

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......

JIF:8.335

Catalase, a therapeutic target in the reversal of estrogen-mediated aging

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......

JIF:8.335

Dichotomous effects of cellular expression of STAT3 on tumor growth of HNSCC

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......

JIF:8.335

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