Parkinson's disease motor symptoms rescue by CRISPRa‐reprogramming astrocytes into GABAergic neurons

J Giehrl‐Schwab, F Giesert, B Rauser… - EMBO molecular …, 2022 - embopress.org
Direct reprogramming based on genetic factors resembles a promising strategy to replace
lost cells in degenerative diseases such as Parkinson's disease. For this, we developed a …

Reprogramming astrocytes to motor neurons by activation of endogenous Ngn2 and Isl1

M Zhou, X Tao, M Sui, M Cui, D Liu, B Wang, T Wang… - Stem Cell Reports, 2021 - cell.com
Central nervous system injury and neurodegenerative diseases cause irreversible loss of
neurons. Overexpression of exogenous specific transcription factors can reprogram somatic …

Precision modulation of neurodegenerative disease-related gene expression in human iPSC-derived neurons

SM Heman-Ackah, AR Bassett, MJA Wood - Scientific reports, 2016 - nature.com
The ability to reprogram adult somatic cells into induced pluripotent stem cells (iPSCs) and
the subsequent development of protocols for their differentiation into disease-relevant cell …

Genome editing in iPSC-based neural systems: from disease models to future therapeutic strategies

A McTague, G Rossignoli, A Ferrini, S Barral… - Frontiers in Genome …, 2021 - frontiersin.org
Therapeutic advances for neurological disorders are challenging due to limited accessibility
of the human central nervous system and incomplete understanding of disease …

Glia-to-neuron conversion by CRISPR-CasRx alleviates symptoms of neurological disease in mice

H Zhou, J Su, X Hu, C Zhou, H Li, Z Chen, Q Xiao… - Cell, 2020 - cell.com
Conversion of glial cells into functional neurons represents a potential therapeutic approach
for replenishing neuronal loss associated with neurodegenerative diseases and brain injury …

CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease

P Pinjala, KP Tryphena, R Prasad, DK Khatri… - Biomaterials …, 2023 - Springer
Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to
correct the causative gene mutation and establish animal models in neurodegenerative …

CRISPR-Cas9-based technology and its relevance to gene editing in Parkinson's disease

MU Rahman, M Bilal, JA Shah, A Kaushik… - Pharmaceutics, 2022 - mdpi.com
Parkinson's disease (PD) and other chronic and debilitating neurodegenerative diseases
(NDs) impose a substantial medical, emotional, and financial burden on individuals and …

FACS-assisted CRISPR-Cas9 genome editing facilitates Parkinson's disease modeling

J Arias-Fuenzalida, J Jarazo, X Qing, J Walter… - Stem Cell Reports, 2017 - cell.com
Genome editing and human induced pluripotent stem cells hold great promise for the
development of isogenic disease models and the correction of disease-associated …

CRISPR and iPSCs: recent developments and future perspectives in neurodegenerative disease modelling, research, and therapeutics

T Sen, RP Thummer - Neurotoxicity Research, 2022 - Springer
Neurodegenerative diseases are prominent causes of pain, suffering, and death worldwide.
Traditional approaches modelling neurodegenerative diseases are deficient, and therefore …

In vivo simultaneous transcriptional activation of multiple genes in the brain using CRISPR–dCas9-activator transgenic mice

H Zhou, J Liu, C Zhou, N Gao, Z Rao, H Li, X Hu… - Nature …, 2018 - nature.com
Despite rapid progresses in the genome-editing field, in vivo simultaneous overexpression
of multiple genes remains challenging. We generated a transgenic mouse using an …