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[HTML][HTML] Recombinant Adeno-Associated Virus vectors for Gene Therapy of the Central Nervous System: delivery routes and clinical aspects
Ż Słyk, N Stachowiak, M Małecki - Biomedicines, 2024 - mdpi.com
The Central Nervous System (CNS) is vulnerable to a range of diseases, including
neurodegenerative and oncological conditions, which present significant treatment …
neurodegenerative and oncological conditions, which present significant treatment …
Targeted CRISPR activation is functional in engineered human pluripotent stem cells but undergoes silencing after differentiation into cardiomyocytes and …
Human induced pluripotent stem cells (hiPSCs) offer opportunities to study human biology
where primary cell types are limited. CRISPR technology allows forward genetic screens …
where primary cell types are limited. CRISPR technology allows forward genetic screens …
Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci
Gene replacement therapies primarily rely on adeno-associated virus (AAV) vectors for
transgene expression. However, episomal expression can decline over time due to vector …
transgene expression. However, episomal expression can decline over time due to vector …
Stable long-term germline transmission of GFP transgenic rat via PiggyBac transposon mediated gene transfer
BJ Jeon, DH Kwon, G Gim, HK Kim, JH Lee… - BMC Veterinary …, 2024 - Springer
Transgene silencing provides a significant challenge in animal model production via gene
engineering using viral vectors or transposons. Selecting an appropriate strategy, contingent …
engineering using viral vectors or transposons. Selecting an appropriate strategy, contingent …
Characterizing a lethal CAG-ACE2 transgenic mouse model for SARS-CoV-2 infection using Cas9-enhanced nanopore sequencing
A Smirnov, A Nurislamov, G Koncevaya, I Serova… - Transgenic …, 2024 - Springer
The SARS-CoV-2 pandemic has underscored the necessity for functional transgenic animal
models for testing. Mouse lines with overexpression of the human receptor ACE2 serve as …
models for testing. Mouse lines with overexpression of the human receptor ACE2 serve as …
Building Blocks of Artificial CRISPR-Based Systems beyond Nucleases
AA Kuzmin, AN Tomilin - International Journal of Molecular Sciences, 2022 - mdpi.com
Tools developed in the fields of genome engineering, precise gene regulation, and synthetic
gene networks have an increasing number of applications. When shared with the scientific …
gene networks have an increasing number of applications. When shared with the scientific …
Methodology to Investigate Protein-Protein Interactions of Jun During Neuronal Differentiation of PC12 Cells
HL Leskinen - 2023 - search.proquest.com
In humans and other mammals, injury to the central nervous system (CNS) can cause a
permanent loss of neuronal function, leading to cognitive defects, limb paralysis, and other …
permanent loss of neuronal function, leading to cognitive defects, limb paralysis, and other …
Development and Validation of a Proximity Labeling Fusion Protein Construct to Identify the Protein–Protein Interactions of Transcription Factors
HL Leskinen, AJ Udvadia - Ocular Regeneration: Methods and Protocols, 2024 - Springer
Dynamic interactions between transcription factors govern changes in gene expression that
mediate changes in cell state accompanying injury response and regeneration …
mediate changes in cell state accompanying injury response and regeneration …
Liver directed lentiviral gene therapy for ARC syndrome
AC Cozmescu - 2023 - discovery.ucl.ac.uk
Arthrogryposis-renal dysfunction-cholestasis (ARC) syndrome is an inherited disorder
typically arising from mutations in the vacuolar protein sorting 33 homolog B (VPS33B) …
typically arising from mutations in the vacuolar protein sorting 33 homolog B (VPS33B) …
Develo** Novel Models and Therapeutics for ZFTA-RELA Ependymoma
S Bonner - 2024 - repository.cam.ac.uk
Paediatric brain tumours are the leading cause of cancer related death in children.
Ependymomas are the third most common brain tumour in children. These tumours are …
Ependymomas are the third most common brain tumour in children. These tumours are …