UK : Gene Therapy used for eye disorder 1st time

UK National Health Service.

Use gene therapy for GENETIC eye disorder for the first time.
Gene Therapy is the obvious 'cure' for FMO3 flaws (or any genetic disorder).

The treatment costs around £600,000 but NHS England has agreed a discounted price with the manufacturer Novartis.
Luxturna (voretigene neparvovec), has been approved by The National Institute for Health and Care Excellence (NICE), which estimates that just under 90 people in England will be eligible for the treatment.

Link to story : BBC News

Another genetic eye disorder patient was treated on NHS last week.

 
Also ...

USA : Base Editing mice with Progeria for 1st time.
New paper.
Gene Therapy in mice with genetic disorder Progeria.
Used the type of gene therapy ... BASE EDITING.
It seems a success.
Link to paper 

Base Editing is for MISSENSE variants in a gene.
Most FMO3 cases will be a MISSENSE variants.
Base Editing is probably safer than replacing the whole gene, but maybe still a while to perfect.
For replacing FMO3 completely, it probably will be by Gene Therapy.

Please remember for this type of therapy, it is currently 'wild west' days.
 

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