FMO3 and CRISPR
A reply was kindly sent by their communications dept about CRISPR in relation to FMO3 gene.
Here is the (abridged) reply :
Dr. Doudna and the Innovative Genomics Institute team appreciate your note. CRISPR genome editing technology is rapidly advancing, but there are still many hurdles to overcome in order to make it safe and effective for a range of genetic diseases. In the case of FMO3 genetic mutations, specific hurdles including the delivery of the CRISPR molecules to the correct cell types, remain difficult. The FMO3 gene appears to be too large to fit into an AAV for delivery into cells via a gene therapy approach, thus genome editing may be the better genetic approach.
It is important to note that it may be years until any genome editing approach to treating FMO3 mutations may be available.
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