Genetical therapy

New CRISPR-based tool inserts large DNA sequences at desired sites in cells

New CRISPR-based tool inserts large DNA sequences at desired sites in cells

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Using the CRISPR gene-editing system, MIT researchers have designed a new tool that can remove faulty genes and replace them with new ones in a safer and more efficient way. Using this system, the researchers showed that they could deliver genes as long as 36,000 DNA base pairs to several types of human cells, as …

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A new family of adeno-associated viral vectors has been shown to be more efficient in delivering genetic cargo

A new family of adeno-associated viral vectors has been shown to be more efficient in delivering genetic cargo

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Gene therapies can treat and even cure some genetic diseases, but it is difficult to deliver the treatments to the parts of the body where they are needed. The researchers engineered viruses called adeno-associated viruses (AAV) to deliver the cargo -; as a functional copy of a gene -; to specific cells and organs, but …

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