QINGYANG ZHANG, SHUXIAN HUANG, DAN WENG^*

BIOCELL, Vol.48, No.10, pp. 1443-1453, 2024, DOI:10.32604/biocell.2024.053794 - 02 October 2024

Abstract Huntington’s disease (HD) is a debilitating neurodegenerative disorder caused by an abnormal expansion of CAG repeats (Cytosine, Adenine, Guanine) in the huntingtin gene (HTT). This mutation leads to the production of a mutant huntingtin protein, resulting in neuronal dysfunction and cell death. Current treatments primarily focus on symptomatic relief and do not address the underlying genetic cause. This review explores spliceosome-mediated RNA trans-splicing (SMaRT) therapy as an innovative and potential approach for HD treatment. SMaRT leverages the cell’s natural splicing machinery to correct mutant mRNA, thereby reducing toxic protein levels while restoring functional protein production. We More >

ELHAM AMJAD¹, RAFFAELE PEZZANI^2,3,*, BABAK SOKOUTI^1,*

Oncology Research, Vol.32, No.3, pp. 439-461, 2024, DOI:10.32604/or.2023.044473 - 06 February 2024

Abstract Noncoding RNAs instruct the Cas9 nuclease to site-specifically cleave DNA in the CRISPR/Cas9 system. Despite the high incidence of hepatocellular carcinoma (HCC), the patient’s outcome is poor. As a result of the emergence of therapeutic resistance in HCC patients, clinicians have faced difficulties in treating such tumor. In addition, CRISPR/Cas9 screens were used to identify genes that improve the clinical response of HCC patients. It is the objective of this article to summarize the current understanding of the use of the CRISPR/Cas9 system for the treatment of cancer, with a particular emphasis on HCC as… More >

MOHAMED AGHYAD AL KABBANI^1,2, GILBERT WUNDERLICH^3,4, CHRISTOPH KöHLER⁵, HANS ZEMPEL^1,2,*

BIOCELL, Vol.46, No.4, pp. 847-853, 2022, DOI:10.32604/biocell.2022.018144 - 15 December 2021

Abstract Tauopathies comprise a spectrum of genetic and sporadic neurodegenerative diseases mainly characterized by the presence of hyperphosphorylated TAU protein aggregations in neurons or glia. Gene therapy, in particular adeno-associated virus (AAV)-based, is an effective medical approach for difficult-to-treat genetic diseases for which there are no convincing traditional therapies, such as tauopathies. Employing AAV-based gene therapy to treat, in particular, genetic tauopathies has many potential therapeutic benefits, but also drawbacks which need to be addressed in order to successfully and efficiently adapt this still unconventional therapy for the various types of tauopathies. In this Viewpoint, we More >

YONGLU WANG^1,2,*, WEI YOU¹, XUEMING LI^3,4,*

BIOCELL, Vol.44, No.2, pp. 167-174, 2020, DOI:10.32604/biocell.2020.09023 - 27 May 2020

Abstract Melanoma is the deadliest type of skin cancer and which has a high ability of metastasis. Surgery is an effective method to treat I or II stage melanoma patients. However, there are few treatment options for metastatic melanoma. Gene therapy is one of the attractive options and is considered as the future direction for treating melanoma. This review mainly discusses the properties and challenges of the various gene therapies in melanoma, especially the delivery systems and gene targeting. More >