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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 terapia genica è un approccio rivoluzionario al trattamento delle malattie genetiche, in cui un trattamento una tantum può fornire benefici per tutta la vita. Invece di trattare solo i sintomi, la terapia genica risolve la malattia alla fonte sostituendo il gene difettoso con una copia sana.

Una copia sana del gene difettoso del paziente viene caricata in un virus che è stato privato del proprio DNA.

Trilioni di virus, ciascuno contenente una copia sana del gene, lo sono  iniettato nel liquido spinale del paziente. 

I virus si legano alle cellule del midollo spinale e del cervello del paziente e forniscono geni sani al nucleo della cellula.

come funziona

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

come funziona

ECHS1 Funding Progress
ECHS1 Fundraising

i nostri partner

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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