Cell and gene therapies hold tremendous value due to their efficacy and potency in a diverse range of indications. The classical approach to gene therapy involves the replacement of a defective gene ...
Cancer is caused by faulty genes, but what also shapes a cancer cell's behavior is how a gene's instructions are trimmed and rearranged before they are turned into the proteins that keep a cell alive.
A growing list of editing tools can be used to make small changes to the genetic code, tweaking key nucleotides to address the underlying causes of diseases. But bigger edits are often needed, and ...