Rett Cures

Education

These videos explain how genetic medicines work. This section comes from Rett.Science.

This is not medical advice. Talk with your doctor.

Genetic medicines are made up of two parts: the cargo which corrects for the mutation, and the delivery technology that gets the cargo into cells.

Cargo

Gene Therapy

more information

Base Editing

Prime Editing

MECP2 Reactivation

RNA Editing and Trans-splicing

Delivery

Viral

Viruses are very efficient at delivering their own genetic material into cells during infection. This function can be harnessed to allow viruses to serve as delivery vehicles for genetic cargos.

Non-Viral

Delivery vehicles extend beyond viruses to include approaches such as lipid nanoparticles and exosomes. Non-viral delivery offers a number of important advantages.

Small RNA

Small RNA are able to self deliver and do not need viral or non viral delivery. They have been used clinically for many years with much success and can therefore advance rapidly into the clinic.