The Giorgio Children's Foundation Raises $350,000 for Neurofibromatosis 1 Research Efforts -- Latest Independent Findings Published in Journal of Clinical Investigation

DUNCANSVILLE, Pa., March 2, 2021 /PRNewswire/ -- The Giorgio Children's Foundation (GCF) announced it had successfully raised over $350,000 in funding for continued Neurofibromatosis Type 1 (NF1) research. NF1 is a genetic disorder which impacts 1 in 3,000 people worldwide and causes tumors to grow on nerves anywhere in the body. Partnering with leading researchers like Dr. David Gutmann of Washington University and Dr. Lu Le of University of Texas Southwestern, GCF continues to be a major proponent of research and education. GCF is a non-profit that has contributed over $2 Million to medical research since 2014.

"We're very pleased with this latest fundraising effort, and extremely excited by the newest NF1 research findings released in the Journal of Clinical Investigation," said Erin Borzellino, Co-Founder of GCF. "Dr. Gutmann and Dr. Le are now using a stem cell engineering method called CRISPR, and were able to develop the first human model of NF-related neurofibromas (tumors), before implanting those models into mice. After finding the cell that initiates tumor development, researchers were able to observe tumor growth in lab mice, showing that they can develop tumors just like humans with NF1. Then, using a specific topical drug, Dr. Le's lab was able to begin shrinking those tumors. This is absolutely game changing. It shows that we are on the right track, and that finding a significant treatment of dermal tumors via medicine is possible. This gives children and adults who are suffering with NF1 some real hope."  

In addition to continued efforts by Dr. Gutmann and Dr. Le, the recently raised funds will go toward a new partnership with Dr Sameer Agnihotri, Ph.D., of University of Pittsburgh Children's Hospital (UPMC Children's). Dr. Agnihotri will focus on gene therapy using innovative methods to correct the mutated NF1 gene, working specifically to address and correct the downstream symptoms caused by the gene mutation.

"I am extremely honored to receive this grant from the Giorgio Foundation, it provides a real boost to our NF1 research," said Dr. Agnihotri, Director of the Brain Tumor Biology and Therapy Lab at UPMC Children's. "Advancing our efforts in gene therapy to stop the growth of NF1 driven tumors could one day lead to curing this painful and disfiguring disease."

For the latest organization events or more information related to NF1, follow GCF on social media: Facebook & Instagram.

About The Giorgio Children's Foundation

A nonprofit organization that supports the research and development of treatments and cures for complications related to NF1, The Giorgio Foundation partners directly with scientists and institutions studying the disease. The Foundation supports efforts specifically focused on and with a proven record of modeling NF1 to achieve real, usable clinical results – ultimately working toward a cure. The Giorgio Foundation also promotes education efforts, shedding light on an often-misunderstood condition, helping patients to achieve a better quality of life because awareness is essential to empathy. Learn more at: www.EndNF1.com.

Media Contact:

Erin Borzellino
1-917-287-9628
[email protected]

SOURCE The Giorgio Children's Foundation