Emily's Entourage atribui uma bolsa de investigação a uma equipa que explora uma nova tecnologia de edição de genes

The project will focus on leveraging the gene editing technology, called prime editing, to correct specific CFTR mutations in a safe and efficient manner and will explore the possibility to deliver this editing cargo through virus-like particles (VLP)

Lower Merion, PA — Nov. 3, 2022 — Emily’s Entourage (EE), an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations, today announced it has awarded $220,000 in funding to Marianne S. Carlon, PhD, and Mattijs Bulcaen of KU Leuven in Belgium to explore the use of a novel gene editing technology, called prime editing, to correct mutations within the cystic fibrosis transmembrane regulator (CFTR) gene in people with CF.

Marianne S. Carlon, PhD, and Mattijs Bulcaen of KU Leuven, will focus on leveraging prime editing to correct specific CFTR mutations in a safe and efficient manner.

Prime editing can write new genetic information into the genome in a very precise manner without causing damage to the host DNA, as no double-strand DNA breaks are induced. A growing body of evidence reports on the versatility, efficiency, and safety of prime editing, but further research is needed to investigate its potential for CF and its ability to correct CFTR mutations. 

Dr. Carlon’s project, titled, “Rewriting drug-refractory CFTR mutations by prime editing,” explores the use of prime editing to correct specific CFTR mutations, the possibility to harness virus-like particles (VLPs) to deliver prime editing in a safe and efficient way, and whether VLPs can efficiently penetrate through CF mucus and enter and gene-edit CF airway epithelial cells.

"At EE we are always eager and excited to fund high-risk, high-reward projects such as prime editing in the hopes of accelerating scientific breakthroughs that might benefit people with CF, especially those in the final 10%,” said Chandra Ghose, PhD, Chief Scientific Officer of Emily’s Entourage. “The importance of funding this grant extends beyond the CF space and may be translatable to other genetic diseases amenable to gene editing.”

This work will inform the CF scientific community on the ability of prime editing to correct mutations within the CFTR gene in a precise manner, as well as to further move VLPs forward as a potential gene delivery strategy.

“The impact of this project lies in advancing knowledge and tools on a prime editing strategy for N1303K within this grant, but with the potential of adapting developed strategies to other CFTR mutations,” said Dr. Carlon. “If gene editing is successful in the future, this offers the perspective of curing CF, thereby improving the quality of life of CF patients.”

Saiba mais sobre as subvenções que a Emily's Entourage financiou em www.emilysentourage.org/awarded-grants. 

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Sobre a comitiva de Emily
A Emily's Entourage é uma organização inovadora 501(c)3 que acelera a investigação de novos tratamentos e de uma cura para os indivíduos no último 10% da população com fibrose quística (FC) que não beneficia das terapias direcionadas para as mutações atualmente disponíveis, incluindo os que têm mutações nonsense da FC. Desde 2011, a Emily's Entourage concedeu milhões de dólares em bolsas de investigação, lançou uma empresa de terapia genética para a FC, desenvolveu um registo de doentes e um programa de combinação de ensaios clínicos para acelerar o recrutamento para ensaios clínicos e liderou esforços a nível mundial para impulsionar a investigação de alto impacto e o desenvolvimento de medicamentos. A organização tem sido referida nos meios de comunicação social nacionais, incluindo o New York Times, STAT, CNN, People, entre outros. Saiba mais em emilysentourage.org.

Contacto para a imprensa
Ashling Knight
Diretor de Marketing e Comunicação
Entourage de Emily
ashling@emilysentourage.org