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Monday, 30 November 2015

The Possible Future Treatment of Sickle cell anaemia republished in geneticdisorders.info

The Possible Future Treatment of Sickle cell anemia"

Source:  "The Possible Future Treatment of Sickle cell anemia"    Tag:  sickle cell anemia pathophysiology at 
 geneticdisorders.info

  
Authors;

* Professor Pranab Kumar Bhattacharya MD(cal) FIC path(Ind)- Professor and Head,* Miss Upasana Bhattacharya- daughter of Prof.PK Bhattacharya   ** Mr. Rupak Bhattacharya Bsc(cal) MSc(JU),** Mr Ritwik Bhattacharya B.com(cal); **Miss Rupsa Bhattacharya** Mr .Soumak Bhattacharya BHM, MSc-student(PUSHA) New Delhi;All of Residence- 7/51 Purbapalli, Sodepur, Dist 24 parganas(north) kol-110, ***Oindrila Mukherjee*** Mrs. Dalia Mukherjee BA(Hons) cal;*** Mr Debasis Mukherjee Bsc(cal).   Dr. Ranu Roy Biswas MD(cal) Asst Professor.  *Dr. Anuradha De DCP(cal) MD(AIIMS) Associate Professor; *Dr. Soma Das MBBS(cal) DCH(cal) Demonstrator; **
·       Affiliation-*   Department of Pathology , Calcutta School of Tropical Medicine, CR Avenue ,Kol-73; W.B; India ** 7/51 Purbapalli, Po-Sodepur; Dist 24 pargnas( West Bengal) Kol-20*** Swamijinagar, South Habra, 24 Parganas(north) West Bengal.

Scientists have learned a great deal about sickle cell anaemia during the past 30 years - what causes it, how it affects the patient, and how to treat some of its complications. They also have begun to have success in developing drugs that will prevent the symptoms of sickle cell anaemia and procedures that should ultimately provide a possible cure.
Some researchers are focusing on identifying drugs that will increase the level of fetal hemoglobin in the blood. studies have shown that these people have less severe cases of the disease. Fetal  hemoglobin seems to prevent 
sickling of red cells, and cells containing fetal hemoglobin tend to  survive longer in the bloodstream.
Hydroxyurea appears to work primarily by stimulating production of fetal hemoglobin. There is some evidence that administering hydroxyurea with erythropoietin, a genetically engineered hormone that stimulates red cell 
production, may make hydroxyurea work more better. This combination approach offers the possibility that lower doses of hydroxyurea can be  used to achieve the needed level of fetal haemoglobin to reduce side effects of both toxic drugs to search for safer agents that are just as effective.'Butyrate,- a simple fatty acid tha is widely used as a food additive, is 
also being investigated as an agent that may increase fetal hemoglobin production. Clotrimazole, an over-the-counter medication commonly used to treat fungal infections, is under investigation as a treatment to prevent the loss of 
water from RBC  that contributes to sickling.  Bone marrow transplantation has been shown to provide a cure for severely     affected children with sickle cell disease. Researchers are working on techniques to  f urther reduce some of the risks of bone marrow transplantation for    patients with sickle cell disease. The ultimate cure for sickle cell anemia may be gene therapy.    Gene therapy offers enormous promise as a potential curative therapy for   SCD, but concerns over the safety of random genomic insertion must first   be resolved . Pre-clinical studies in mice have provided the proof of    principle that transduction of bone marrow stem cells with lentiviral   vectors that express a beta-globin gene can prevent Hb S polymerization in  vivo. The wide range of abnormalities engendered by the sickle cell  mutation offers several other opportunities for therapeutic interventions. For example, the NIH Road Map is supporting ongoing investigations in   which high-through put screening approaches are used to discover novel low  -molecular-weight compounds that can alter key aspects of the disease,  I ncluding hemoglobin polymerization, expression of Hb F, and leukocyte    adhesion. Current clinical trials are evaluating the efficacy of Ca2+-  sensitive Gardos channel inhibitors , with or without hydroxyurea, in    preventing dehydration of erythrocytes . Vasoactive drugs (e.g., NO,   sildenafil, endothelin antagonists) are being evaluated for the treatment  i f pulmonary hypertension. Statins are of potentially great interest since they can increase NO production and reduce leukocyte adhesion . Selective antagonists &  Intravenous   gammaglobulins are currently under clinical evaluation following a study   demonstrating a dose-dependent reduction in leukocyte adhesion and in the   number of interactions between rbc and wbc, accompanied by improvements inmicrocirculatory blood flow and survival of sickle transgenic mice . Almost a century after SCD was first 
described, we may be towards the dawn of a new era in which a physician one day might be able to use genetic information to select one or more drugs that target specific aspects of disease pathophysiology that are 
relevant to a particular patient with SCD 


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