Lentiviral vectors for delivery of gene-editing systems based on CRISPR/Cas: current state and perspectives

W Dong, B Kantor - Viruses, 2021 - mdpi.com
CRISPR/Cas technology has revolutionized the fields of the genome-and epigenome-
editing by supplying unparalleled control over genomic sequences and expression …

CRISPR/Cas9-based application for cancer therapy: challenges and solutions for non-viral delivery

YQ Lin, KK Feng, JY Lu, JQ Le, WL Li, BC Zhang… - Journal of Controlled …, 2023 - Elsevier
CRISPR/Cas9 genome editing is a promising therapeutic technique, which makes precise
and rapid gene editing technology possible on account of its high sensitivity and efficiency …

The mechanistic role of alpha-synuclein in the nucleus: impaired nuclear function caused by familial Parkinson's disease SNCA mutations

V Chen, M Moncalvo, D Tringali… - Human molecular …, 2020 - academic.oup.com
Alpha-synuclein SNCA has been implicated in the etiology of Parkinson's disease (PD);
however, the normal function of alpha-synuclein protein and the pathway that mediates its …

Multiangle Light Scattering as a Lentivirus Purification Process Analytical Technology

SA Sripada, E Barbieri, S Shastry… - Analytical …, 2024 - ACS Publications
The limited biomolecular and functional stability of lentiviral vectors (LVVs) for cell therapy
poses the need for analytical tools that can monitor their titers and activity throughout the …

Therapeutic modulation of gene expression in the disease state: Treatment strategies and approaches for the development of next-generation of the epigenetic drugs

J Rittiner, M Cumaran, S Malhotra… - … in Bioengineering and …, 2022 - frontiersin.org
Epigenetic dysregulation is an important determinant of many pathological conditions and
diseases. Designer molecules that can specifically target endogenous DNA sequences …

Neuronal-type-specific epigenome editing to decrease SNCA expression: Implications for precision medicine in synucleinopathies

Z Sun, B Kantor, O Chiba-Falek - Molecular Therapy-Nucleic Acids, 2024 - cell.com
Overexpression of SNCA has been implicated in the pathogenesis of synucleinopathies,
particularly Parkinson's disease (PD) and dementia with Lewy bodies (DLB). While PD and …

Ultrasound-Assisted CRISPRi-Exosome for Epigenetic Modification of α-Synuclein Gene in a Mouse Model of Parkinson's Disease

W Kong, X Li, X Guo, Y Sun, W Chai, Y Chang… - ACS …, 2024 - ACS Publications
Currently, there is a lack of effective treatment for Parkinson's disease (PD). In PD patients,
aberrant methylation of SNCA (α-synuclein gene) has been reported and may be a potential …

[HTML][HTML] An improved protocol for the production of lentiviral vectors

LY Brown, W Dong, B Kantor - STAR protocols, 2020 - Elsevier
Lentiviral vectors are an ideal gene-delivery system for large gene-editing tools, such as the
clustered regularly interspaced short palindromic repeat (CRISPR)-Cas9 system, due to …

Research and therapeutic approaches in stem cell genome editing by CRISPR toolkit

B Mollashahi, H Latifi-Navid, I Owliaee, S Shamdani… - Molecules, 2023 - mdpi.com
The most widely used genome editing toolkit is CRISPR (clustered regularly interspaced
short palindromic repeats). It provides the possibility of replacing and modifying DNA and …

Delivery platforms for CRISPR/Cas9 genome editing of glial cells in the central nervous system

V Meneghini, M Peviani, M Luciani… - Frontiers in Genome …, 2021 - frontiersin.org
Glial cells (astrocytes, oligodendrocytes, and microglia) are emerging as key players in
several physiological and pathological processes of the central nervous system (CNS) …