Our Research

Gene Regulation Therapy

Gene Regulation Therapy

We have named our approach to curing sickle cell disease Gene Regulation Therapy, and the result is called a phenotypic cure. That is to say, no genes are permanently altered; instead, we use normal bodily signals to turn off the sickle cell gene and activate another gene with the same function in its place. We have discovered a naturally occurring protein, a protein that is grown in our bodies that will shut off the sickle cell gene. This same protein will also turn on a fetal hemoglobin gene to replace the silenced sickle hemoglobin. There are a few people in the world in which this substitution of fetal hemoglobin for sickle hemoglobin occurs naturally, and these people never have the devastating effects of sickle cell disease or any other physical problems. Our recent experiments with blood from sickle cell patients have shown that this approach will work as predicted. The treatment should be easy to deliver, as a protein directly into the blood or marrow, where red cell precursors have receptors on their surface that recognize this protein and internalize it. We have also discovered a plant compound that will activate expression of this protein in human cells, and the plant compound will be tested for both safety and effectiveness in humans with sickle cell or beta-thalassemia.

Comments

  1. Hi Dr. Broyles,

    I have a daughter with Sickle Cell Disease SS. I would like to know more about this breakthrough and how far the general population is away from being able to recieve Gene Regulation Therapy. You mentioned this would have to occur a few times per year…would this have to be an on-going therapy for the remainder of ones life? I’d like to be contacted concerning this breakthrough.

    Thank you!

  2. Robert H. Broyles, PhD says:

    Reply to Sharice (Sept 3):

    Dear Sharice,
    I hope that your daughter has good medical care, at least to the extent possible for SCD.
    We do not know exactly how far away we are from making our treatment generally available. We must first find a financial partner who will help us conduct clinical trials, and we do not know how long it will take to obtain the necessary funds. We hope to start clinical trials in a year from now, but that is optimistic that we will find the necessary partners soon. Please urge your friends to donate through PayPal (very secure) on this website by clicking on the gold Donate button, and donate yourself if you can afford to do that.

    Yes, this treatment will have to be given several times a year; and it will have to be taken for the rest of the patient’s life. But “the rest of the patient’s life” will then become a normal life span; and the pain should become a thing of the past. The treatments will be very inexpensive, about 10% of what it now costs for inferior, partially effective treatment. And there should be no side effects.

    All the best,

    Robert H. Broyles, Ph.D.
    President, SCCF

  3. Am one of this ss and am currently taken my post graduate course in Biochemistry specializing in Gene Regulatory Therapy. Please can you send me articles on it? I will appreciate.

  4. God bless you and your team.i would really like time when d treament will start being administered to patient.and how soon can it get to nigeria.it’s for my brother who is SS.i have lost two siblings already due to sickle cell disease.hope to hear from you soon.tanx and Godbless

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