Medical oncology (Northwood, London, England)
Dec 26, 2025
Adeno-associated virus (AAV) has emerged as a pivotal vector for cancer gene therapy due to its low immunogenicity, non-pathogenicity, and sustained transgene expression capacity. However, the heterogeneity and complexity of the tumor microenvironmen...
Adeno-associated virus (AAV) vectors are excellent gene-delivery carriers in gene therapy; however, improperly packaged capsids produced during manufacturing can reduce potency and raise safety concerns. We introduce a machine-learning-assisted, low-...
Small ruminant lentiviruses (SRLV) are responsible for significant economic losses in sheep and goat farming; however, effective vaccination strategies remain unavailable. This study evaluated the immunogenicity, safety, and protective efficacy of a ...
Serotonin neurons are central to the pathophysiology and therapeutics of mental disorders, including major depressive disorder, anxiety, and schizophrenia. Genetically modified mice make it possible to target serotonin neurons by selective expression...
Adeno-associated virus (AAV)-based therapeutics have the potential to transform the lives of patients by delivering one-time treatments for a variety of diseases. However, a critical challenge to their widespread adoption and distribution is the high...
Molecular therapy : the journal of the American Society of Gene Therapy
Apr 1, 2025
Adeno-associated virus (AAV) has emerged as a highly promising vector for human gene therapy due to its favorable safety profile, versatility, and ability to transduce a wide range of tissues. However, natural AAV serotypes have shortcomings, includi...
Advanced science (Weinheim, Baden-Wurttemberg, Germany)
Feb 11, 2025
Adeno-associated virus (AAV) has emerged as a leading vector for gene therapy due to its broad host range, low pathogenicity, and ability to facilitate long-term gene expression. However, AAV vectors face limitations, including immunogenicity and ins...
The design of CRISPR gRNAs requires accurate on-target efficiency predictions, which demand high-quality gRNA activity data and efficient modeling. To advance, we here report on the generation of on-target gRNA activity data for 10,592 SpCas9 gRNAs. ...
A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors. Recent advances in high-throughput DNA synthesis, mu...
Modern experimental technologies can assay large numbers of biological sequences, but engineered protein libraries rarely exceed the sequence diversity of natural protein families. Machine learning (ML) models trained directly on experimental data wi...
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