Results 191 to 200 of about 116,785 (352)

Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection

open access: gold
Hao Liu   +19 more
openalex   +1 more source

Adeno-Associated Virus-Based Gene Therapy for Lafora Disease in <i>Epm2b</i>-Deficient Mice. [PDF]

open access: yesInt J Mol Sci
Zafra-Puerta L   +6 more
europepmc   +1 more source

Intravenous Adeno-Associated Virus Serotype 8 Encoding Urocortin-2 Provides Sustained Augmentation of Left Ventricular Function in Mice

open access: green, 2013
Mei Hua Gao   +14 more
openalex   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

open access: yesBiotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai   +9 more
wiley   +1 more source

A case study showing the role of hydrophobicity variants and other enriched mAb proteoforms on filterability through a virus filter with productivity improvement measures

open access: yesBiotechnology Progress, EarlyView.
Abstract A rapid assessment of manufacturability for drug candidates is crucial for advancing a prospective biotherapeutic from a candidate to a bulk drug substance. A lot‐to‐lot approach to manufacturability is adopted where each biologic batch is assessed for manufacturability as a bulk, unfractionated pool.
Solomon Isu   +6 more
wiley   +1 more source

TULP4, a novel E3 ligase gene, participates in neuronal migration as a candidate in schizophrenia

open access: yesCNS Neuroscience &Therapeutics, EarlyView., 2023
Mutations identified from four SCZ pedigrees resulted in decreased TULP4 expression. Tulp4 knockdown caused delayed neuron migration in embryonic mice, and impaired cognition and prepulse inhibition in adult mice. These phenotypes may be related to TULP4 through its involvement in the formation of a novel E3 ubiquitin ligases.
Yan Bi   +19 more
wiley   +1 more source

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