Coverage of our work & advancements in developing gene therapies to treat neurodegenerative conditions.
In the News
There are significant advancements being made in the field of gene replacement therapy, including for diseases impacting the Central Nervous System.
More than 400 gene therapy trials are currently underway in the United States.
A Brief History of Riaan Research Initiative
Riaan Research Initiative partners with Rarebase to identify drugs that may assist patients with Cockayne Syndrome, a fatal neurodegenerative genetic disorder.
Riaan Research Initiative funds Cockayne Syndrome gene replacement therapy research at UMass Chan Medical School
"The partnership of the Medical School's gene therapy team with the Riaan Research Initiative will lead to a first-in-human AAV gene therapy clinical trial for Cockayne Syndrome as fast as humanly possible. Children like Riaan inspire us to work harder every day to overcome and persist through the challenges in research to give them a change at a better life."
— Miguel Sena-Esteves, PhD, UMass Chan Medical School
Recent progress and considerations for AAV gene therapies targeting the central nervous system
Current Clinical Applications of In Vivo Gene Therapy with AAVs
IN THE NEWS
My Son Was Born With An Incredibly Rare Disease & Fighting for His Life Isn’t Anything Like I Imagined
Personal Reflections on Riaan & Riaan Research Initiative: Listen Here
We have already achieved the impossible': fighting a rare childhood disease with hope.
Interview with RRI Founder, Jo Kaur
Scientists Partially Restored a Blind Man’s Sight With New Gene Therapy
An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future
First Gene Therapy for Tay-Sachs Disease Successfully Given to Two Children
Central Utah toddler makes remarkable progress after groundbreaking gene replacement therapy
Our inspiration Riaan caught two (!) viruses from
Gene therapy for Cockayne syndrome: a synopsis! (C
Happy Mother's Day to all moms, and especially to
Happy Mother's Day! Meet some of our CS moms. Che
From our inspiration Riaan's mom. What fun songs d
"These are the moments I want to live in. I’m in
Eid Mubarak to all of our friends and supporters w
A picture is worth a thousand words. A facial expr
(Part 2/2) Caroline Bologna wrote a piece for The
(Part 1/2) Caroline Bologna wrote a piece for The
A huge thanks to everyone who attended our Cockayn
Our Cockayne syndrome drug repurposing webinar is
Meet Rui! There are families all over our world
"HOW WE GOT HERE" (LISTEN TO THE FULL SPEECH - L
Our inspiration Riaan, standing tall and proud in
Our kids work hard, at school, in life, and every
A special shout out to our hero, our inspiration -
Join us for a free webinar on April 6, 2023 to lea
Today is the second anniversary of our inspiration
Our inspiration Riaan during his first day of pre-
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