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.
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
Ducks, Monkeys, and Alligators! We are always on a
Happy 29 months to our joyful, resilient, larger t
No parent should ever have to join this club. To t
Happy Mother's Day! Check out our latest blog post
How much are their smiles worth? #CockayneSyndrom
Every day, from morning to night, can be exhaustin
#CureCockayneSyndrome #CockayneSyndrome #ERCC8 #ER
For such a small patient population, we have a lot
With Mother's Day 2022 around the corner, we are l
The original beach boy. #Run4Riaan #CureCockayneS
Congratulations, Richie, on finishing the half-mar
A message from Riaan's dad, Richie, who will be ru
All ready for the race tomorrow! Good luck Richie.
The race is coming up! Get your donations in! Spre
Read the full interview at the link in our bio. (I
Check out this interview we did with the amazing @
Riaan enjoys watching his dad get ready for the ra
Getting ready for the race! Riaan's dad test drive
We are pleased to announce the launch of the #Cock
Wishing all who celebrate a joyous time, and wishi
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