Help Advance Gene Therapy for NF2 on Rare Disease Day!
$2,212.31
Raised
18
Donations
$20,000
Goal
Help Advance Gene Therapy for NF2 on Rare Disease Day!
Join us in marking Rare Disease Day by supporting NF2 BioSolutions' mission to advance gene therapy for NF2! Your donation will directly fund groundbreaking research led by the Meyer Lab at Nationwide Children's Hospital. Together, we can move closer to clinical trials and bring hope to individuals battling NF2 worldwide. Every dollar counts in our fight against this rare genetic disorder. Let's make a difference together!
Donate now and be a part of the solution!
🔍 The Research:
The groundbreaking Gene Therapy led by Dr. Meyer at Nationwide Children's Hospital focuses on restoring the Merlin protein in the NF2 gene by using an AAV9 viral construct as a delivery mechanism. The efficacy of this construct is currently undergoing testing. Testing using intratumoral injections is also ongoing in the KO (Knockout) mouse and xenograft mouse models. We successfully showed the expression of merlin in tumor cells, accompanied by reduced tumor growth in the xenograft model.
Pre-IND documents explaining our clinical trial plan are being prepared to be submitted to the FDA. We have also applied for a National Institute of Health (NIH) grant to fund the preparation of a future trial.
💞 Join Us on Rare Disease Day:
Let's make a difference together. Please help us get closer to finding a cure for NF2. We're all in this fight together, and your support means the world to us! If you'd like to have your fundraiser that you can share with your friends and family, contact us at rebeca-chajon@nf2biosolutions.org.
Our donors
ML
M. Loizzo
donated
$20
Per guarire e continuare ba sperare
BE
Benjamin Eckman
donated
$1,000
DW
Dee White
donated
£20