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
Thank you! #GivingTuesday
Wowza! We received a very generous $15,000 anonymo
[GIVING TUESDAY MATCH] This Giving Tuesday, we are
Tomorrow is GIVING TUESDAY, the international day
We are thankful for: - The scientists working on
A huge and hearty thank you to all 36 donors who s
DASVANDH NETWORK MATCHING CAMPAIGN UPDATE! $3,65
A good start to Dasvandh Week! Thank you to everyo
The Dasvandh Network (DVN) matching fund LAUNCHES
We have a new matching campaign that will launch F
Today our team met with the gene therapy scientist
As parents of children with a lethal rare disease,
This Halloween, Chef Riaan is cookin' up a cure! T
October! Our inspiration Riaan enjoys pumpkin seas
From all of us here at Riaan Research Initiative,
For love, for siblings we hope will be together fo
It's been a big period of transition and growing u
Our inspiration Riaan meets his little brother, Ji
Read our latest post, "The Night Before Labor," at
Today is the International Day of Sign Languages!
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