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ECHS1 Research



Your support is crucial in raising awareness and funds for gene therapy pre-clinical research, preparation for a Toxicology Study, or whatever exciting initiatives may be next. 

The sooner we reach our goals, the faster we get to a potential Clinical Trial.

See our Fundraising Progress below.

Thank you for your SUPPORT

Total Updated May 2, 2024

 La thérapie génique est une approche révolutionnaire du traitement des maladies génétiques, où un traitement ponctuel peut offrir des avantages à vie. Au lieu de traiter uniquement les symptômes, la thérapie génique corrige la maladie à la source en remplaçant le gène défectueux par une copie saine.

Une copie saine du gène défectueux du patient est chargée dans un virus qui a été dépouillé de son propre ADN.

Des milliards de virus, chacun contenant une copie saine du gène, sont  injecté dans le liquide céphalo-rachidien du patient. 

Les virus se lient aux cellules de la moelle épinière et du cerveau du patient et transmettent des gènes sains au noyau de la cellule.

Comment ça fonctionne

ECHS1 Funding Progress

the gene therapy project

Several years ago, a group of ECHS1 families forged a partnership with researchers in the Bailey Lab at UT Southwestern Medical Ceneter, located in Dallas, TX, USA.


The Bailey Lab is working on the use of an adeno-associated viral (AAV) vector for therapeutic gene delivery.

the bailey lab team

Dr. Rachel Bailey is an Assistant Professor in Pediatric Neurology with a secondary appointment in the Center for Alzheimer's and Neurodegenerative Diseases.

Meghan Eller is a Neuroscience Graduate Student and

Madison Bunce is a Green Fellow.

the teaM AT WORK

Comment ça fonctionne

ECHS1 Funding Progress
ECHS1 Fundraising

nos partenaires

It truly takes a village.


Families from around the world are partnering with Cure Mito Foundation to raise funds that get us closer to a treatment or cure for our children.


If you are interested in supporting a fund which honors a child or family member affected by ECHS1 Deficiency, please click the "DONATE" button associated with one of the campaigns below.

Please email if you would like information about starting a fund in honor or memory of your loved one.

Thank you for supporting our children!


Team Lila - ECHS1

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Team Richie - Fighting for a Cure for ECHS1

Team Nora.jpg

Team Nora - ECHS1

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ECHS1 Logo.png
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