Autologous Cd34+ Enriched Cell Fraction That Contains Cd34+ Cells Transduced With Retroviral Vector That Encodes For The Human Ada Cdna Sequence
A gene therapy product in which a patient's own blood stem cells are genetically modified to carry a working copy of the ADA gene, then returned to the patient to restore the immune system in ADA-SCID. This personalized approach aims to provide a lasting cure by correcting the underlying genetic defect.
กลไกการออกฤทธิ์
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
HBD / HBA
- / -
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
คำถามที่พบบ่อย
A gene therapy product in which a patient's own blood stem cells are genetically modified to carry a working copy of the ADA gene, then returned to the patient to restore the immune system in ADA-SCID. This personalized approach aims to provide a lasting cure by correcting the underlying genetic defect.
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
Yes, Autologous Cd34+ Enriched Cell Fraction That Contains Cd34+ Cells Transduced With Retroviral Vector That Encodes For The Human Ada Cdna Sequence is an approved drug. It has reached clinical phase 4. It is classified as a Unknown.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL5315093. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
ข้อจำกัดความรับผิดชอบทางการแพทย์
This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.
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