Results 191 to 200 of about 116,785 (352)
Adeno-Associated Virus-Based Gene Therapy for Lafora Disease in <i>Epm2b</i>-Deficient Mice. [PDF]
Zafra-Puerta L +6 more
europepmc +1 more source
Suppressing microtubule detyrosination augments adeno-associated virus 2 endosomal escape and gene delivery. [PDF]
Tripathi S +5 more
europepmc +1 more source
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
Heterogeneity in an adeno-associated virus transfection-based production process limits the production efficiency. [PDF]
Ladd B +5 more
europepmc +1 more source
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
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
Insights into Adeno-Associated Virus Capsid Charge Heterogeneity. [PDF]
Wu J, Draper BE, Jarrold MF, Heger CD.
europepmc +1 more source

