Sickle Cell Anemia — Hope from Gene Therapy – Medical Informer Video

This inherited red blood cell disorder is the most common genetic disease in America, and one of the most painful and debilitating. Until now, bone marrow and cord blood transplants have been the only treatment available to patients, and matched donors are hard to find. But an experiment at Genetix in Cambridge, Massachusetts is raising hopes. By treating the bone marrow of mice with anti-sickling gene therapy, scientists saw great improvement in normal red blood production.All 50 Secrets of the Sequence videos have an accompanying classroom-tested lesson that encourages students to further explore the video topics. Each lesson includes background information, state and national science standards, discussion questions and answers, teacher notes and an activity that will ensure a hands-on, “minds-on” experience. To see lessons for this series, visit
Video Rating: 4 / 5

13 Responses to “Sickle Cell Anemia — Hope from Gene Therapy – Medical Informer Video”

  1. rikhobra says:

    So i read here that 72 Patients are undergoing trail by the therepy as mentioened in the video here. can nay one tell the status of those 72 SCD patients.
    When will this therapy be avaliable for commom man.

    I have a sone 3 and a half year ols son who is having this problem. it is difficult to see him inthe problem.

  2. STFUNOOBZZ says:

    There is a possibility. He may carry ‘the sickle-cell anemia trait’, which means that he carries the gene for this genetic defect. This means will give the trait to his children (which means that his kids will also carry the risk of having children with sickle cell anemia). If he has children with another person with sickle cell anemia, his children will most likely have sickle cell anemia. Hope this helps :)

  3. Harsh Savalia says:

    Quick question for those who know their genetics. Will Chris’s kids have sickle cell even though he has been treated for it?

  4. SufferInJuly says:

    Cancer is a disease, a chronic metabolic disease akin to other chronic metabolic diseases such as scurvy, night blindness, pellagra & pernicious anemia. No such disease has ever been cured through mechanical manipulation of the body. Where cancer is seated isn’t relevant to its cure. The preventative, cure & treatment of this vitamin-deficiency disease is Amygdalin (vitamin B17). People who get at least 100 m.g. of B17 daily cannot contract cancer. It works for sickle cell anemia too.

  5. eagleeye2391 says:

    my ex gf has sickle cell

  6. Shaela69 says:

    I am 2 out of 4 kids. My older sister and the youngest of my little brothers have sickle cell anemia. I have sickle cell trait and my other brother doesnt have either.

  7. xBattleHeartx says:

    Sickle cell anemia VIRUS! FTW! Come on Government scientists!!

  8. thecrazywierdo14 says:

    i have a friend with sickle cell anemia

  9. drerror says:

    May your brother RIP :)

  10. BioGuelph says:

    The hardest thing in gene therapy was never selecting the right gene, but finding or making the right virus to act as your vector. If you get it wrong the treatment could easily kill the patient or they may start developing cancerous cells. Luckily vector testing techniques are improving every day, and targeting only specified tissues.

  11. drjj10 says:

    reaserch may be proceding right as we type, but i am sure that it won’t b available for a while. once they test it on mice doesn’t mean it will work right away on humans. but there is hope!!

  12. 2468Shock says:

    Humans have 2 Cells suppose to be 2 Normal Cells. Anemia people have 1 cell Alpha Anemia, 1 Cell Beta lower Anemia Cell or Thalasemia which is a Lower Anemia. A Person with Anemia & a Normal cell will live past 80 years a Anemia & Thalasemia will live to 15 to 28.

  13. webothgowrongx33 says:

    4:00 That is the cutest baby EVER!


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