Related topics: cells · mouse model · virus · genes · cancer cells

Machine-learning how to create better AAV gene delivery vehicles

Adeno-associated viruses (AAVs) have become promising vehicles for delivering gene therapies to defective tissues in the human body because they are non-pathogenic and can transfer therapeutic DNA into target cells. However, ...

Microneedles for therapeutic gene delivery

There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections. The therapies work by delivering genes into the patients' cells, which then ...

Scientists update genome editing technology

Researchers from Peter the Great St. Petersburg Polytechnic University (SPbPU) in collaboration with colleagues from the Pavlov University, ITMO University, and the University of Hamburg compared their developed carriers ...

Editing immune response could make gene therapy more effective

Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In the case of CRISPR-based gene therapies, molecular scissors can then snip out a defective gene, add in a missing ...

New understanding of CRISPR-Cas9 tool could improve gene editing

Within a mere eight years, CRISPR-Cas9 has become the go-to genome editor for both basic research and gene therapy. But CRISPR-Cas9 also has spawned other potentially powerful DNA manipulation tools that could help fix genetic ...

Simulations show how to make gene therapy more effective

Diseases with a genetic cause could, in theory, be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human cells is difficult. A promising method for the delivery ...

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Gene therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

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