Viral vector‐based gene therapies in the clinic

Z Zhao, AC Anselmo, S Mitragotri - … & translational medicine, 2022 - Wiley Online Library
Gene therapies are currently one of the most investigated therapeutic modalities in both the
preclinical and clinical settings and have shown promise in treating a diverse spectrum of …

The approved gene therapy drugs worldwide: from 1998 to 2019

CC Ma, ZL Wang, T Xu, ZY He, YQ Wei - Biotechnology advances, 2020 - Elsevier
With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): where are we, and how did we get here?

AM Keeler, TR Flotte - Annual review of virology, 2019 - annualreviews.org
The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Inflammatory cytokines in cancer: comprehensive understanding and clinical progress in gene therapy

T Lan, L Chen, X Wei - Cells, 2021 - mdpi.com
The relationship between chronic inflammation and neoplastic diseases is not fully
understood. The inflammatory microenvironment of a tumor is an intricate network that …

[PDF][PDF] A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing

A Edraki, A Mir, R Ibraheim, I Gainetdinov, Y Yoon… - Molecular cell, 2019 - cell.com
CRISPR-Cas9 genome editing has transformed biotechnology and therapeutics. However,
in vivo applications of some Cas9s are hindered by large size (limiting delivery by adeno …

Viral delivery systems for CRISPR

CL Xu, MZC Ruan, VB Mahajan, SH Tsang - Viruses, 2019 - mdpi.com
The frontiers of precision medicine have been revolutionized by the development of
Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing …

microRNAs in Parkinson's disease: from pathogenesis to novel diagnostic and therapeutic approaches

L Leggio, S Vivarelli, F L'Episcopo, C Tirolo… - International journal of …, 2017 - mdpi.com
Parkinson's disease (PD) is the most prevalent central nervous system (CNS) movement
disorder and the second most common neurodegenerative disease overall. PD is …

[HTML][HTML] Non-viral in vitro gene delivery: it is now time to set the bar!

N Bono, F Ponti, D Mantovani, G Candiani - Pharmaceutics, 2020 - mdpi.com
Transfection by means of non-viral gene delivery vectors is the cornerstone of modern gene
delivery. Despite the resources poured into the development of ever more effective …

Recent advances in stimulus‐responsive nanocarriers for gene therapy

C Yu, L Li, P Hu, Y Yang, W Wei, X Deng… - Advanced …, 2021 - Wiley Online Library
Gene therapy provides a promising strategy for curing monogenetic disorders and complex
diseases. However, there are challenges associated with the use of viral delivery vectors …

Biomolecular ultrasound and sonogenetics

D Maresca, A Lakshmanan, M Abedi… - Annual review of …, 2018 - annualreviews.org
Visualizing and modulating molecular and cellular processes occurring deep within living
organisms is fundamental to our study of basic biology and disease. Currently, the most …