Leer en: Español

Stem Cells in Focus

An Exciting Strategy for Treating Sickle Cell Disease with Stem Cells

Stem cell researchers are getting closer to a new treatment for sickle cell disease, moving promising laboratory research into human clinical trials. Millions of people worldwide suffer from this hereditary disease.

Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules, which can distort red blood cells into a sickle shape. The sickled cells break down prematurely causing anemia. The sickled cells can also get stuck in small blood vessels depriving the tissues and organs of oxygen-rich blood. The illustration to the right shows a healthy, round, red blood cell next to a sickled cell.

Symptoms of sickle cell disease include anemia (a low number of red blood cells), severe pain, difficulty breathing, strokes, joint pain and arthritis, blockage of blood flow in the spleen or liver and severe infections.

Sickle cell disease is caused by an inherited mutation in the beta-globin gene, and it is here at the source that stem cell scientists are targeting their efforts. Hematopoietic stem cells generate all the cells of the blood and immune system. If the mutation could be corrected in these stem cells, all the red blood cells arising from the repaired stem cells would carry the normal beta-globin and function normally.

Dr. Donald Kohn of UCLA and has demonstrated this approach is feasible by removing the stem cells from the bone marrow of people with sickle cell disease and precisely correcting the genetic mutation in the hemoglobin gene. In the laboratory, these corrected hematopoietic stem cells make normal red blood cells. Ultimately, the goal is to transplant the repaired stem cells back into patients with sickle cell disease. Dr. Kohn and his team are one step closer to this goal having received approval for a phase 1 clinical trial (May 2015) that will assess the safety and initial evidence of effectiveness of treating sickle cell disease patients by transplanting them with their own genetically corrected hematopoietic stem cells. Learn more about clinical trials here.

Sickle cell disease can currently be treated with the transplantation of bone marrow from a healthy donor, which provides a source of new blood-forming stem cells which produce normal red blood cells. However, bone marrow transplantation is dependent on a well-matched donor and comes with the risk that the recipient’s body may reject the transplanted cells. Dr. Kohn’s strategy has the potential to provide a more effective and safe treatment.

The California Institute for Regenerative Medicine, which is funding the clinical trial, illustrates the treatment strategy in its newest Stem Cells in Your Face video:


Leave a comment
  1. james jack | Feb 15, 2018
    Make the most of mainly premium substances - you will find him or her for: Car Title Loans Orlando
  2. teena | Dec 30, 2017
    i like viewing web sites which comprehend the price of delivering the excellent useful resource free of charge. I truly adored reading your posting free minecraft gift card generator
  3. Sahil | Dec 29, 2017
  4. bhai log | Dec 15, 2017
    This is a great discovery, I hope it will entail many good ideas and opportunities free gift card codes generator
  5. inforr | Dec 08, 2017
    I was reading the provided article with a great interest! This post is useful and informative free gift code generator
  6. dedi galih wisnumurti | Nov 22, 2017
    Keep up the excellent work , I read few articles on this site and I think that your web blog is real interesting and Power to the People of excellent information.1. Jasa SEO2. Google SEO3. Pembuatan Website4. Google Adwords5. SEO6. WebsiteJasa SEOGoogle AdwordsPembuatan Website7. Sewa Villa di Puncak8. Villa di Puncak
  7. nancywilsonvp | Nov 22, 2017
    Nice post, thanks for sharing this. We provide Dell Customer Service.If you have any issues related to Dell Laptop then visit here:Dell Support UK
  8. rara | Nov 17, 2017
  9. rara | Nov 17, 2017
  10. haha | Nov 17, 2017
  11. wawa | Nov 17, 2017
  12. rara | Nov 17, 2017
  13. pavina | Nov 16, 2017
    incredible substance and truly interesting.We are truly fortunate to discover such blog with astounding substance free paypal gift code generator 
  14. dedi galih wisnumurti | Nov 16, 2017
    Nice post. I learn something more challenging on distinct blogs everyday. It will always be stimulating to read content off their writers and practice a little something from their store. I’d choose to use some with all the content in my small weblog whether you do not mind. Natually I’ll provide a link on your own internet weblog. Many thanks sharing.1. Jasa SEO2. Jasa SEO3. Google SEO4. Jasa Pembuatan WebsiteDAN INI TUKANG NYEPAM KAMI1. Sewa Villa Di Puncak2. Villa Puncak
  15. Toshia Trieu | Oct 26, 2017
    Memiliki daya tahan yang baik terhadap panas, karat dan goresan/gesekan – Tahan temperature rendah maupun tinggi – Memiliki kekuatan besar dengan massa yang kecil – Keras, liat, densitasnya besar dan permukaannya tahan aus  Toko Plat Bordes Toko Plat Bordes Pabrik bondek Toko Wiremesh Besi Pabrik besi unp undangan pernikahan cantik Distributor Besi Hollow undangan pernikahan simple Jual Besi Hollow Baja Toko besi hollow Distributor besi beton Sni Ulir Polos Agen Plat Bordes Agen Plat Bordes jual hp asus murah jual hp asus murah Pabrik Plat kapal besi baja bki krakatau steel Agen Wiremesh Besi Pabrik besi siku undangan pernikahan simple Distributor Wiremesh Harga Besi Hollow Baja jual laptop asus jual laptop asus jual laptop asus murah jual laptop asus murah Agen besi hollow Distributor besi beton ais Pabrik Plat Bordes Pabrik Plat Bordes Pabrik besi siku Pabrik Wiremesh Besi Pabrik Plat besi plat kapal undangan pernikahan mewah Distributor Besi Pipa Baja Schedule 80 Pabrik Besi Hollow Baja Pabrik besi hollow Distributor besi beton Distributor Wiremesh Besi Distributor Wiremesh Besi Pabrik pipa besi baja sch 40 sch 80 Distributor Atap Spandek Pabrik besi h beam baja undangan pernikahan cute Distributor Besi Pipa Baja Schedule 40 Distributor Besi Beton Distributor pipa besi jasa konstruksi baja Harga Wiremesh Besi Harga Wiremesh Besi Pabrik Plat besi Harga Atap Spandek Pabrik besi cnp undangan pernikahan modern undangan pernikahan online undangan pernikahan biru Distributor Pipa Besi Hitam Medium Supplier Besi Beton Harga pipa besi www.pusatbesibaja.co.id Jual Wiremesh Besi Jual Wiremesh Besi Pabrik besi h beam Jual Atap Spandek www.gudangbesibaja.com undangan pernikahan buatan sendiri Distributor Besi Wf Agen Besi Beton Jual pipa besi www.pusatbesibaja.com Supplier Wiremesh Besi Supplier Wiremesh Besi Pabrik besi wf Supplier Atap Spandek Distributor besi Wf Baja undangan pernikahan bahasa inggris islam Distributor Besi H beam Toko Besi Beton Supplier pipa besi www.jualbesibajamurah.com Toko Wiremesh Besi Toko Wiremesh Besi Pabrik wiremesh Toko Atap Spandek Distributor bondek undangan pernikahan amplop surat Distributor Besi Beton Jual Besi Beton Toko pipa besi Jual besi beton Sni Ulir Polos polos ulir Agen Wiremesh Besi Agen Wiremesh Besi Pabrik besi unp Agen Atap Spandek undangan pernikahan undangan pernikahan amplop surat Distributor wiremesh undangan pernikahan unik dan murah www.sentrabesibaja.com Harga Besi Beton Agen pipa besi Jual besi beton sii Pabrik Wiremesh Besi Pabrik Wiremesh Besi Pabrik besi cnp Pabrik Atap Spandek Distributor pipa besi baja sch 40 sch 80 undangan pernikahan elegan Jual Welded Beam T beam King Queen Cross Castellated Beam Pabrik Besi Beton Pabrik pipa besi Jual besi beton psi Ulir Polos Distributor Atap Spandek Distributor Atap Spandek Pabrik besi hollow Distributor Bondek Cor Distributor besi hollow undangan pernikahan animasi Jual Bondek Cor Distributor Besi Wf Baja Distributor sch 40 Jual besi beton perwira Harga Atap Spandek Harga Atap Spandek Pabrik besi beton Sni Ulir Polos Harga Bondek Cor Distributor besi beton Sni Ulir Polos undangan pernikahan lucu Jual Atap Spandek Sni Supplier Besi Wf Baja Harga sch 40 Jual besi beton PAS Jual Atap Spandek Jual Atap Spandek ironsteelcenter.com Jual Bondek Cor Distributor besi wf baja undangan pernikahan kalender Jual Plat Bordes Kembang Agen Besi Wf Baja Jual sch 40 Jual besi beton master steel ms Supplier Atap Spandek Supplier Atap Spandek Distributor stainless steel Supplier Bondek Cor Distributor besi unp undangan pernikahan ala india Jual Plat Besi Hitam Toko Besi Wf Baja Supplier sch 40 Jual besi beton ksty Toko Atap Spandek Toko Atap Spandek Distributor atap spandek Toko Bondek Cor Distributor besi siku undangan pernikahan klasik Jual Plat Kapal Bki Krakatau Steel Jual Besi Wf Baja Toko sch 40 Jual besi beton krakatau steel ks link5 

    Leave a comment