Results 251 to 260 of about 116,785 (352)

CRISPR Enabled Precision Oncology: From Gene Editing to Tumor Microenvironment Remodeling

Med Research, EarlyView.
CRISPR technology has progressed from a prokaryotic immune system to a diverse suite of editing platforms, including Cas nucleases, base and prime editors, and RNA‐targeting enzymes. These advances enable precise genomic and epigenomic interventions, high‐throughput functional screening, and immune engineering.
Kailai Li, Peixin Huang, Yue Qian, Anqi Lin, Jingjun He, Junyi Shen, Li Chen, Kai Miao, Jian Zhang   +8 more
wiley   +1 more source

M/G-Ratio-Tuned Calcium Alginate Hydrogel Microcarrier for Controlled Delivery of Adeno-Associated Virus for Gene Therapy. [PDF]

Biotechnol Bioeng
Hioki A, Takatsuka S, Kurashina Y, Onoe H.   +3 more
europepmc   +1 more source

My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective

Human Gene Therapy, 2020
openaire   +2 more sources

Nuclear Alpha‐Synuclein: Mechanisms and Implications for Synucleinopathies

Movement Disorders, EarlyView.
Abstract Alpha‐synuclein (aSyn), historically studied for its synaptic functions and central role in Lewy body pathology, is emerging as a protein with significant nuclear activities relevant to Parkinson's disease (PD) and related synucleinopathies. Recent advances reveal that aSyn dynamically localizes to neuronal nuclei in both health and disease ...
Tiago Fleming Outeiro, David J. Koss
wiley   +1 more source

Development of cell-based assay for detecting replication-competent adeno-associated virus by qPCR. [PDF]

Mol Ther Methods Clin Dev
Shevade A, Reeves JS, Bak H, Tustian AD.
europepmc   +1 more source

Uncovering the Complexity of Synucleinopathies: An Ongoing Tale Between Proteins and Lipids

Movement Disorders, EarlyView.
Abstract Neurodegenerative diseases are pathological states characterized by progressive alterations in brain homeostasis during aging. Synucleinopathies, including Parkinson's disease and dementia with Lewy bodies, are defined neuropathologically by the accumulation of inclusions known as Lewy bodies and Lewy neurites.
Manuel Flores‐León, Tiago F. Outeiro
wiley   +1 more source

Advances in gene therapy for Lafora disease: Intravenous recombinant adeno-associated virus-mediated delivery of EPM2A and EPM2B genes. [PDF]

Clin Transl Med
Zafra-Puerta L, Iglesias-Cabeza N, Sciaccaluga M, Bellingacci L, Canonichesi J, Sánchez-Martín G, Costa C, Sánchez MP, Serratosa JM.   +8 more
europepmc   +1 more source

The Expanding Role of Gene Sequencing in Shaping Fetal Therapies: Clinical and Ethical Considerations

Prenatal Diagnosis, EarlyView.
ABSTRACT In utero interventions are transformative in addressing genetic and anatomic conditions during fetal development. Next generation sequencing enables early genetic testing, playing a pivotal role in prenatal decision‐making by supporting risk stratification, precise and timely diagnosis, which directly informs eligibility for fetal surgical and
Matthew A. Shear, Beltran Borges, Billie R. Lianoglou, Tony Lum, Emma Canepa, Jennifer L. Cohen, Julia E. H. Brown, Akos Herzeg   +7 more
wiley   +1 more source

Rescue of <i>Iqsec2</i> Knockout Mice with Human IQSEC2 Adeno-Associated Virus Mediated Gene Therapy. [PDF]

Int J Mol Sci
Soundararajan D, Kouyama-Suzuki E, Shirai Y, Orth S, Borisov V, Israel Y, Weiss Y, Avi-Isaac L, Garoma NH, Lache O, Levy NS, Li S, Zang W, Netser S, Wagner S, Jimenez G, Frankel WN, Tabuchi K, Sands TT, Levy AP.   +19 more
europepmc   +1 more source

AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease) [PDF]

, 2018
et al,, Jiang, Xuntain, Ory, Daniel S
core   +1 more source