Reverse Rett

The Time is Now

Gene therapy attacks Rett Syndrome at its root by replacing an unhealthy gene with a healthy one and could not only treat multiple symptoms but may ultimately lead to a cure for Rett. The RSRT Gene Therapy Consortium is now completing its 3rd year working towards this goal. We want to help them.

history Campaign has now closed

It ran from 12:00 PM, 29 November 2016 to 12:01 PM, 6 December 2016

Registered Charity in England and Wales (1136809)

open_in_new http://www.reverserett.org.uk
Check mark Match funded

Campaign target

£50,000

Amount raised

£105,616

Donations

142

    Categories

  • Health/WellbeingHealth/Wellbeing
  • Medical ResearchMedical Research

    Helping

Location

Situation

Rett Syndrome strikes at random in early childhood, affecting little girls almost exclusively. Each of these girls will lose the ability to walk, talk and use their hands. Most will have difficulty breathing and eating. All are at increased risk of sudden and unexplained death. Many girls live into adulthood, requiring total, 24-hour-a-day care. There is currently no effective treatment but the disease has already been reversed in a mouse model.

Solution

The concept behind gene therapy is simple: deliver a healthy gene to compensate for one that is mutated. It has already been proven that a single one-time administration of a gene therapeutic can restore health in the mouse model of this disease. RSRT’s Gene Therapy Consortium is a collaboration of four labs that are in their third year of working together to develop effective methods to use Gene Therapy to reverse Rett Syndrome in people.

  • While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett it is conceivable that gene therapy can correct the cause of Rett at its very source and thus provide a profound recovery of function.

    — Stuart Cobb

  • We‚Äôve learned that a single one-time administration of a gene therapeutic can have a clinically meaningful result in the workhorse rodent model of this disease, even when delivered later in life.

    — Brian Kaspar

  • The openness of the investigators propels our studies and makes for a productive venture that would not be possible by any one individual laboratory.

    — Gail Mandel

  • While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett it is conceivable that gene therapy can correct the cause of Rett at its very source and thus provide a profound recovery of function.

    — Stuart Cobb

  • We’ve learned that a single one-time administration of a gene therapeutic can have a clinically meaningful result in the workhorse rodent model of this disease, even when delivered later in life.

    — Brian Kaspar

  • The openness of the investigators propels our studies and makes for a productive venture that would not be possible by any one individual laboratory.

    — Gail Mandel