Krabbe Disease

- Cell Therapy Approaches

Krabbe disease is caused by mutations in the GALC gene, resulting in galactocerebrosidase deficiency. Impaired metabolism results in lipid accumulation that affects the growth of the myelin sheaths and causes severe degeneration of motor skills.

Cell Therapy Approaches

Hematopoietic stem cell (HSC) transplantation is a potential treatment for Krabbe disease, which appears to be beneficial in late-onset cases or in infant patients diagnosed before or at birth. The most significant benefit was demonstrated in asymptomatic or mildly symptomatic patients and when transplanted within the first month of life.

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LifeMap Discovery

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Krabbe Disease

- Cell Therapy Approaches

Cell Therapy Approaches

Cell Therapies for the treatment of

Modeling of

Affected or Therapy-targeted Cells/Compartments for

Literature related to

Welcome to LifeMap Discovery

Watch the introductory video to learn more about our database of embryonic development for stem cell research and regenerative medicine

Krabbe Disease - Cell Therapy Approaches

Cell Therapy Approaches

Cell Therapies for the treatment of

Modeling of

Affected or Therapy-targeted Cells/Compartments for

Literature related to

Welcome to
LifeMap Discovery

Krabbe Disease

- Cell Therapy Approaches