Yao Y, Bei F
Adv Neurobiol. 2024; 41:91-112.
PMID: 39589711
DOI: 10.1007/978-3-031-69188-1_4.
Gosai S, Castro R, Fuentes N, Butts J, Mouri K, Alasoadura M
Nature. 2024; 634(8036):1211-1220.
PMID: 39443793
PMC: 11525185.
DOI: 10.1038/s41586-024-08070-z.
Unda S, Pomeranz L, Marongiu R, Yu X, Kelly L, Hassanzadeh G
Sci Adv. 2024; 10(41):eadp9150.
PMID: 39383230
PMC: 11463271.
DOI: 10.1126/sciadv.adp9150.
Zhang Y, Zhu Z, Li Z, Feng J, Long J, Deng Y
Cell Biosci. 2024; 14(1):125.
PMID: 39343943
PMC: 11441009.
DOI: 10.1186/s13578-024-01296-4.
Moaveni A, Amiri M, Shademan B, Farhadi A, Behroozi J, Nourazarian A
Front Mol Biosci. 2024; 11:1382190.
PMID: 38836106
PMC: 11149429.
DOI: 10.3389/fmolb.2024.1382190.
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Massaro G, Geard A, Nelvagal H, Gore K, Clemo N, Waddington S
Hum Mol Genet. 2024; 33(17):1467-1480.
PMID: 38757200
PMC: 11336133.
DOI: 10.1093/hmg/ddae081.
Precise Gene Knock-In Tools with Minimized Risk of DSBs: A Trend for Gene Manipulation.
Liu Y, Kong J, Liu G, Li Z, Xiao Y
Adv Sci (Weinh). 2024; 11(28):e2401797.
PMID: 38728624
PMC: 11267366.
DOI: 10.1002/advs.202401797.
Targeting the chromatin structural changes of antitumor immunity.
Li N, Lun D, Gong N, Meng G, Du X, Wang H
J Pharm Anal. 2024; 14(4):100905.
PMID: 38665224
PMC: 11043877.
DOI: 10.1016/j.jpha.2023.11.012.
Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of Leber congenital amaurosis.
Liu Y, Tai J, Yu C, Xu D, Xiao D, Pang J
Front Med (Lausanne). 2024; 10:1291795.
PMID: 38264046
PMC: 10803578.
DOI: 10.3389/fmed.2023.1291795.
Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.
Henderson M, Zieba J, Li X, Campbell D, Williams M, Vogt D
BioTech (Basel). 2024; 13(1).
PMID: 38247731
PMC: 10801589.
DOI: 10.3390/biotech13010001.
Machine-guided design of synthetic cell type-specific -regulatory elements.
Gosai S, Castro R, Fuentes N, Butts J, Kales S, Noche R
bioRxiv. 2023; .
PMID: 37609287
PMC: 10441439.
DOI: 10.1101/2023.08.08.552077.
Bidirectional Regulation of Motor Circuits Using Magnetogenetic Gene Therapy Short: Magnetogenetic Regulation of Motor Circuits.
Unda S, Pomeranz L, Marongiu R, Yu X, Kelly L, Hassanzadeh G
bioRxiv. 2023; .
PMID: 37503198
PMC: 10369996.
DOI: 10.1101/2023.07.13.548699.
Recessive aminoacyl-tRNA synthetase disorders: lessons learned from disease models.
Kalotay E, Klugmann M, Housley G, Frohlich D
Front Neurosci. 2023; 17:1182874.
PMID: 37274208
PMC: 10234152.
DOI: 10.3389/fnins.2023.1182874.
Molecular and cognitive signatures of ageing partially restored through synthetic delivery of IL2 to the brain.
Lemaitre P, Tareen S, Pasciuto E, Mascali L, Martirosyan A, Callaerts-Vegh Z
EMBO Mol Med. 2023; 15(5):e16805.
PMID: 36975362
PMC: 10165365.
DOI: 10.15252/emmm.202216805.
AAV Engineering for Improving Tropism to the Central Nervous System.
Ghauri M, Ou L
Biology (Basel). 2023; 12(2).
PMID: 36829465
PMC: 9953251.
DOI: 10.3390/biology12020186.
Electroacupuncture of the trigeminal nerve causes -methyl-D-aspartate receptors to mediate blood-brain barrier opening and induces neuronal excitatory changes.
Gong P, Zhang S, Ren L, Zhang J, Zhao Y, Mao X
Front Cell Neurosci. 2022; 16:1020644.
PMID: 36313622
PMC: 9606778.
DOI: 10.3389/fncel.2022.1020644.
Transduction of Systemically Administered Adeno-Associated Virus in the Colonic Enteric Nervous System and c-Kit Cells of Adult Mice.
Wang L, Yuan P, Challis C, Kumar S, Tache Y
Front Neuroanat. 2022; 16:884280.
PMID: 35734536
PMC: 9207206.
DOI: 10.3389/fnana.2022.884280.
Rescue of behavioral and electrophysiological phenotypes in a Pitt-Hopkins syndrome mouse model by genetic restoration of expression.
Kim H, Gao E, Draper A, Berens N, Vihma H, Zhang X
Elife. 2022; 11.
PMID: 35535852
PMC: 9090324.
DOI: 10.7554/eLife.72290.
Gene therapy of yeast NDI1 on mitochondrial complex I dysfunction in rotenone-induced Parkinson's disease models in vitro and vivo.
Li H, Sun B, Huang Y, Zhang J, Xu X, Shen Y
Mol Med. 2022; 28(1):29.
PMID: 35255803
PMC: 8900322.
DOI: 10.1186/s10020-022-00456-x.
Gene Therapy to the Liver and Nervous System: Promises and Challenges.
Cantore A, Fraldi A, Meneghini V, Gritti A
Front Med (Lausanne). 2022; 8:774618.
PMID: 35118085
PMC: 8803894.
DOI: 10.3389/fmed.2021.774618.