...hope on the horizon for Sickle Cell disease sufferer? Nevertheless, there is immunity from malaria.
P.S. Sickle Cell disease, a congenital haemolytic anaemia causing terrible body ache upon exacerbation by clogging blood vessels, is due to polymerisation of mutated sickle Haemoglobin (HbS - Glutamic acid replaced by Valine at position 6: Normal adult - HbA and normal fetal - HbF). HbF doesn't allow polymerisation to happen but whatever little remains after infancy, is inhibited by DNMT1 (DNA Methyltransferase 1).
A new FDA approved drug, Decitabine (Dec) can deplete DNA methyltransferase (DNMT1) and to prolong its action oral Tetrahydrouridine (THU), a CDA inhibitor (Cytidine Deaminase inhibitor) is added.
Thus, combining oral Dec with oral THU, the THU-Dec combination is showing promising results in the phase 1 clinical trial.
Potential New Therapy on Horizon for Sickle Cell Disease: https://consultqd.clevelandclinic.org/2014/12/ash-sickle-cell/amp/
Sickle-cell disease:
https://en.m.wikipedia.org/wiki/Sickle-cell_disease
P.S. Sickle Cell disease, a congenital haemolytic anaemia causing terrible body ache upon exacerbation by clogging blood vessels, is due to polymerisation of mutated sickle Haemoglobin (HbS - Glutamic acid replaced by Valine at position 6: Normal adult - HbA and normal fetal - HbF). HbF doesn't allow polymerisation to happen but whatever little remains after infancy, is inhibited by DNMT1 (DNA Methyltransferase 1).
A new FDA approved drug, Decitabine (Dec) can deplete DNA methyltransferase (DNMT1) and to prolong its action oral Tetrahydrouridine (THU), a CDA inhibitor (Cytidine Deaminase inhibitor) is added.
Thus, combining oral Dec with oral THU, the THU-Dec combination is showing promising results in the phase 1 clinical trial.
Potential New Therapy on Horizon for Sickle Cell Disease: https://consultqd.clevelandclinic.org/2014/12/ash-sickle-cell/amp/
Sickle-cell disease:
https://en.m.wikipedia.org/wiki/Sickle-cell_disease
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