Restoring gene function by the induced skipping of deleterious exons has been shown to be effective for treating genetic disorders. However, many of the clinically successful therapies for exon skipping are transient oligonucleotide-based treatments ...
Drug repurposing has attracted increased attention, especially in the context of drug discovery rates that remain too low despite a recent wave of approvals for biological therapeutics (e.g. gene therapy). These new biological entities-based treatmen...
Over 100 000 research articles and 9000 patents have been published on tissue engineering (TE) in the past 20 years. Yet, very few TE products have made their way to the market during the same period. Experts have proposed a variety of strategies to ...
The advancement of cell injections has created a need for accurate, efficient, and low-invasive injections. However, the conventional approaches to reduce cell damage during penetration, mainly optimization of micropipette tips and vision-based autom...
As artificial intelligence (AI), machine learning (ML) and other digital tools gain prominence across industries, their application in cell and gene therapy (CGT) has become a topic of increasing interest. Discussions at recent meetings of the Intern...
Adeno-associated virus (AAV) is a non-enveloped DNA virus infecting a wide variety of species, tissues, and cell types, which is recognized as a safe and effective method for delivering therapeutic transgenes. AAV vector is the most popular viral gen...
Investigative ophthalmology & visual science
Jan 2, 2025
PURPOSE: To quantify outer retina structural changes and define novel biomarkers of inherited retinal degeneration associated with biallelic mutations in RPE65 (RPE65-IRD) in patients before and after subretinal gene augmentation therapy with voretig...
Adrenoleukodystrophy is a genetic metabolic disorder characterized by a heterogeneous phenotype. Its severe form, known as cerebral adrenoleukodystrophy, involves unpredictable cerebral damage and progressive central nervous system deterioration. Thi...
Target gene delivery is crucial to gene therapy. Adeno-associated virus (AAV) has emerged as a primary gene therapy vector due to its broad host range, long-term expression, and low pathogenicity. However, AAV vectors have some limitations, such as i...
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