Results 71 to 80 of about 144,727 (384)
When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of the infusate and the region targeted. We have previously shown that AAV is disseminated within
Ernesto A Salegio+12 more
doaj +1 more source
NLO evolution kernels for skewed transversity distributions [PDF]
We present a calculation of the two-loop evolution kernels of the twist-two transversally polarized quark and linearly polarized gluon skewed parton distributions in the minimal subtraction scheme and discuss a solution of the evolution equations ...
A. Freund+41 more
core +4 more sources
Recent Applications of Mesoporous Silica Nanoparticles in Gene Therapy
The review summarizes the synthesis of mesoporous silica nanoparticles (MSNs) with modifiable surface properties, functionalization strategies, mechanism of therapeutic payload release, and current applications in gene therapy, focusing on their capabilities in the targeted delivery of therapeutic nucleic acids, CRISPR‐Cas systems, and other genetic ...
Tamanna Binte Huq+4 more
wiley +1 more source
Mutations affecting the BSCL2 gene cause the most severe form of congenital generalised lipodystrophy (CGL). Affected individuals develop severe metabolic complications including diabetes and hepatic steatosis.
George D. Mcilroy+4 more
doaj +1 more source
High levels of AAV vector integration into CRISPR-induced DNA breaks
Adeno-associated virus (AAV) vectors have shown promising results in preclinical models, but the genomic consequences of transduction with AAV vectors encoding CRISPR-Cas nucleases is still being examined.
Killian S. Hanlon+19 more
semanticscholar +1 more source
AAV and Insertional Mutagenesis
In this issue of Molecular Therapy, David Russell provides an invited commentary on data presented at the tenth annual meeting of the American Society of Gene Therapy that strongly suggested that adenovirus-associated virus (AAV) vector insertion could in certain models be linked to the development of cancer.1 A short report describing the data that ...
openaire +3 more sources
Lymphoid and CXCR4 Cell Targeted Lipid Nanoparticles Facilitate HIV‐1 Proviral DNA Excision
Advancements in ART improve HIV‐1 patient outcomes but are unable to eliminate latent viral DNA. To address this, CXCR4‐targeted lipid nanoparticles (T‐LNPs) for delivering CRISPR‐Cas9 to excise HIV‐1 DNA in infected cells are developed. These T‐LNPs achieve ≈60% HIV‐1 DNA excision efficacy in blood and splenic tissue, demonstrating promise for ...
Sudipta Panja+6 more
wiley +1 more source
Here we present a novel tracing technique to stain projection neurons in Golgi-like detail by double viral infection. We used retrograde lentiviral vectors and adeno-associated viral vectors (AAV) to drive TET-ON/TET-OFF system in neurons connecting two ...
Akiya eWatakabe+11 more
doaj +1 more source
Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model
Significance In humans, inner ear development is completed in utero, with hearing onset at ∼20 weeks of gestation. However, genetic forms of congenital deafness are typically diagnosed during the neonatal period.
O. Akil+12 more
semanticscholar +1 more source
Convergence drawing is used to create flexible, microscale, multifunctional fiber‐based neural probes. Optimized materials selection enables individual devices to perform neural recording, electrical stimulation, optogenetics, fiber photometry, fluid delivery, and voltammetric neurotransmitter detection in rodents.
Nicolette Driscoll+16 more
wiley +1 more source