Results 151 to 160 of about 32,863 (259)

Enhancing hemophilia A gene therapy by strategic F8 deletions in AAV vectors. [PDF]

Blood Sci
Zhao JJ, Tian SN, Li X, Peng ZY, Li GH, Zhang F, Zhao M, Zhang JP, Zhang XB.   +8 more
europepmc   +1 more source

Spatial tropism profiling of AAV vectors by ultrasensitive sequential FISH in tissue. [PDF]

Nat Biotechnol, 2023
europepmc   +1 more source

Establishing Sensory Neurons as Therapeutic Targets in Peripheral Neuropathy Driven by Polyglutamine Expanded Murine ATXN3

Annals of Neurology, EarlyView.
Repeat expansion disorders frequently involve peripheral neuropathy, yet mechanisms remain unclear. Using a spinocerebellar ataxia type 3 (SCA3) Knock‐In Atxn3Q300/Q6, we identify progressive sensorimotor deficits, peripheral nerve pathology, and dorsal root ganglia RNA splicing dysregulation.
Juan P. Mato, John M. Hayes, Jacen Emerson, Maeha Sridharan, Toluwani Adelaja, Arsal Naeem, Alexandra F. Putka, Hayley S. McLoughlin   +7 more
wiley   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA. [PDF]

Mol Ther Methods Clin Dev
Khan SA, Álvarez JV, Nidhi FNU, Benincore-Florez E, Tomatsu S.   +4 more
europepmc   +1 more source

What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?

Biotechnology and Bioengineering, EarlyView.
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady, Nicholas Donohue, Alexandra Bogdanovic, Sharon Davin, Brian Glennon, Niall Barron, C. Mark Smales   +6 more
wiley   +1 more source

Multistep allelic conversion in mouse pre-implantation embryos by AAV vectors. [PDF]

Sci Rep
Nickl P, Jenickova I, Elias J, Kasparek P, Barinka C, Kopkanova J, Sedlacek R.   +6 more
europepmc   +1 more source

Evaluating the transduction efficiency of systemically delivered AAV vectors in the rat nervous system. [PDF]

Front Neurosci, 2023
Yang OJ, Robilotto GL, Alom F, Alemán K, Devulapally K, Morris A, Mickle AD.   +6 more
europepmc   +1 more source

Fouling‐Free Inertial Microfluidic Cell Clarification for Lentivirus Manufacturing

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Lentiviral vectors (LVVs) are widely used as gene‐delivery vehicles in cell and gene therapy, enabling stable integration of transgenes into target cells. However, efficient clarification of LVV harvest remains a major bottleneck in large‐scale manufacturing, as conventional depth filtration often results in significant product loss.
Dohyun Park, Alexander Bevacqua, Mingyang Cui, Marina Nichols, Jianzhu Chen, Jongyoon Han   +5 more
wiley   +1 more source

Double-strand break repair pathways differentially affect processing and transduction by dual AAV vectors. [PDF]

Nat Commun
Maurer AC, Benyamini B, Whitney ON, Fan VB, Cattoglio C, Kissiov DU, Dailey GM, Darzacq X, Weitzman MD, Tjian R.   +9 more
europepmc   +1 more source