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
Sarah Matthews and Brett Colley, the incredible pa
Check out our latest post on Substack! "From the
We are so excited to celebrate the fifth birthday
There's no joy like an empty bowl! CS parents and
As the fictional character Ted Lasso would advise
Look at the difference gene therapy can make! Noa
Today on National Grief Awareness Day, we are rais
Today is National Grief Awareness Day. Cockayne s
We wanted to extend a heartfelt thank you to every
Winty shares his reflections after finishing the t
Winty is approaching the finish line in Vancouver!
Instagram post 17967887321409285
Winty talks about Cockayne syndrome and the work o
From our founder Jo Kaur: "Yesterday, I was grabb
Winty is 75 miles in to his 2 day fundraising ride
Winty crossed mile 35 over an hour ago! The total
Today's the day! Winty Singh just started his ride
In a recent piece for Insider, author Melissa Monr
Raise your hand if you donated to #Ride4Riaan and/
A message from our founder, Jo Kaur: "Today, ten
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