TERAPIA DE GENES
HELP US REACH OUR CURRENT $1.5M GOAL!
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.
ā
See our Fundraising Progress below.
ā
Thank you for your SUPPORT
La terapia gĆ©nica es un enfoque revolucionario para el tratamiento de enfermedades genĆ©ticas, donde un tratamiento Ćŗnico puede brindar beneficios de por vida. En lugar de tratar solo los sĆntomas, la terapia gĆ©nica soluciona la enfermedad en su origen al reemplazar el gen defectuoso con una copia sana.
Una copia sana del gen defectuoso del paciente se carga en un virus al que se le ha quitado su propio ADN.
Trillones de virus, cada uno de los cuales contiene una copia sana del gen, se inyectado en el lĆquido cefalorraquĆdeo del paciente.
Los virus se unen a las cĆ©lulas en la mĆ©dula espinal y el cerebro del paciente y entregan genes saludables al nĆŗcleo de la cĆ©lula.
cĆ³mo funciona
nuestros compaƱeros
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.
ā
Please email info@curemito.org if you would like information about starting a fund in honor or memory of your loved one.
ā
Thank you for supporting our children!
