Thalassemia Patients and Friends
Discussion Forums => Miscellaneous Questions => Topic started by: Dori on September 22, 2009, 09:47:12 AM
-
Genetically modified stem cells for curing erythrocyte pyruvate kinase deficiency http://tinyurl.com/npzcrg
Link: http://twitter.com/sciclips/status/4070659846
Q1) What are stem cells?
Q2) Will this help PK (def) patients?
I have retweet them and send it to the three pk def people I speak the most. I know I should know what stem cells are, but I am confused at the moment.
Dore
p.s. I've done some more research. I am not sure, There are a lot of strange followers behind this one. Some followers look not "clean". :huh
-
This is the real article:
http://www.ncbi.nlm.nih.gov/pubmed/19755962?ordinalpos=15&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum
Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme.
Meza NW, Alonso-Ferrero ME, Navarro S, Quintana-Bustamante O, Valeri A, Garcia-Gomez M, Bueren JA, Bautista JM, Segovia JC.
[1] Hematopoiesis and Gene Therapy Division. Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) y Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBER-ER), Madrid, Spain [2] LABIEMET, School of Medicine of Táchira, Universidad de los Andes, San Cristóbal, Venezuela.
Human erythrocyte R-type pyruvate kinase deficiency (PKD) is a disorder caused by mutations in the PKLR gene that produces chronic nonspherocytic hemolytic anemia. Besides periodic blood transfusion and splenectomy, severe cases require bone marrow (BM) transplant, which makes this disease a good candidate for gene therapy. Here, the normal human R-type pyruvate kinase (hRPK) complementary (cDNA) was expressed in hematopoietic stem cells (HSCs) derived from pklr deficient mice, using a retroviral vector system. These mice show a similar red blood cell phenotype to that observed in human PKD. Transduced HSCs were transplanted into myeloablated adult PKD mice or in utero injected into nonconditioned PKD fetuses. In the myeloablated recipients, the hematological manifestations of PKD were completely resolved and normal percentages of late erythroid progenitors, reticulocyte and erythrocyte counts, hemoglobin levels and erythrocyte biochemistry were restored. Corrected cells preserved their rescuing capacity after secondary and tertiary transplant. When corrected cells were in utero transplanted, partial correction of the erythrocyte disease was obtained, although a very low number of corrected cells became engrafted, suggesting a different efficiency of cell therapy applied in utero. Our data suggest that transduction of human RPK cDNA in PKLR mutated HSCs could be an effective strategy in severe cases of PKD.
-
All the best for the people with PKD , i hope n wish that there will b a secure and potential cure for PKD ...
Best REgards
Take Care
Umair
-
Unfortunately not for my PKD. But thankfully for another Pkd which is 10000x worse than me if not progressive. It's :-( since this step is more little than a babystep.
-
Dore,
I hope that research like this leads to a cure for you some day very soon.
Sharmin