Muscular dystrophy (MD) comprises a class of genetic conditions characterized by the progressive degeneration and weakness of skeletal muscle. Genetic etiologies differ among the major muscular dystrophies: myotonic dystrophy type 1 (DM1) is linked t...
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...
Alzheimer's disease (AD), acknowledged as the leading cause of dementia, is defined by the accumulation of amyloid plaques and neurofibrillary tangles (NFTs) in the brain. This condition presents a significant challenge to global health due to its co...
Journal of controlled release : official journal of the Controlled Release Society
Nov 27, 2025
Glioblastoma (GBM) is an aggressive central nervous system (CNS) malignancy with a poor prognosis and limited responses to conventional therapies. RNA-based therapeutics, with their gene-targeting specificity, present a promising avenue for GBM treat...
CRISPR-based epigenetic editing enables reversible regulation of gene expression without permanent DNA modification. The integration of artificial intelligence (AI) enhances guide RNA (gRNA) design, off-target prediction, and delivery optimization. W...
Developmental and epileptic encephalopathies (DEEs) are severe neurological disorders characterized by childhood-onset seizures and significant developmental impairments. Seizures are often refractory to treatment with traditional antiseizure medicat...
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...
Ocular gene therapy offers a promising approach for treating various eye diseases, centered on the process of transfection, including delivery, cellular uptake and gene expression. This study addresses anatomical and physiological barriers, such as t...
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...
PURPOSE: To use artificial intelligence (AI) for quantifying schisis volume (ASV) in X-linked retinoschisis (XLRS) for use as a structural endpoint in gene therapy clinical trials.
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