Successful gene therapy carried out on a patient with Sickle cell disease
Sickle cell disease is among the most prevalent inherited genetic disorders,about 90,000 people in the United States alone have sickle cell disease.Gene therapy may provide a long term and curative treatment for patients with the disease.
Gene therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
According to a report published in the March issue of the New England Journal of Medicine,a clinical trial was carried out on 15 years old boy in May 2014 and was administered the LentiGlobin(Blue bird bio,inc.)gene therapy in October 2014.
The patient's bone marrow was conditioned with Busulfan which provides room for re-population and was enriched for CD34-stem cells.
The researchers who reported at the American Hematology Society meeting in December 2016 said red cell transfusion continued until the transplanted cells began to produce altered Hb ceasing on day 88.
By the ninth month,post-transplant levels of HbA were 5.5g/dL(46%)increasing to 5.7g/dL(48%)by the fifteenth month.By the eighteenth month mark,HbA levels had risen to 6.6g/dL(53%).At the same time,levels of HbS fell.
At twelve months,the proportion of sickled cells in the patient and his oxygen saturation levels were similar to those of his mother,a Heterozygote.Proportions of all blood cell types had normalized.
Also,a second research team testing the same gene therapy reported their findings at the American Hematology Society meeting stating that their seven patients have shown more modest improvements.
The importance of this approach includes,lower risk of rejection compared with allogeneic transplant,there is no need for a donor and just one treatment that could lead to a permanent cure.
According to Dr.Steven J.Gary,PhD from the gene therapy center at the university of North Carolina,Chapel Hill:
Dr.Gary designed the viral vector for the giant axonal neuropathy gene therapy clinical trial and others.The experimental gene therapy took place at the Necker Children's hospital in Paris.
Dr.Philippe Leboulch an author of the research and Professor of Medicine said:
Dr.Trish Wong of Oregon Health and Science university says,the new study is truly amazing work,proof of principle that a cure for this chronic,devastating disease is in sight.
Gene therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
According to a report published in the March issue of the New England Journal of Medicine,a clinical trial was carried out on 15 years old boy in May 2014 and was administered the LentiGlobin(Blue bird bio,inc.)gene therapy in October 2014.
The patient's bone marrow was conditioned with Busulfan which provides room for re-population and was enriched for CD34-stem cells.
The researchers who reported at the American Hematology Society meeting in December 2016 said red cell transfusion continued until the transplanted cells began to produce altered Hb ceasing on day 88.
By the ninth month,post-transplant levels of HbA were 5.5g/dL(46%)increasing to 5.7g/dL(48%)by the fifteenth month.By the eighteenth month mark,HbA levels had risen to 6.6g/dL(53%).At the same time,levels of HbS fell.
At twelve months,the proportion of sickled cells in the patient and his oxygen saturation levels were similar to those of his mother,a Heterozygote.Proportions of all blood cell types had normalized.
Also,a second research team testing the same gene therapy reported their findings at the American Hematology Society meeting stating that their seven patients have shown more modest improvements.
The importance of this approach includes,lower risk of rejection compared with allogeneic transplant,there is no need for a donor and just one treatment that could lead to a permanent cure.
According to Dr.Steven J.Gary,PhD from the gene therapy center at the university of North Carolina,Chapel Hill:
''This is one of the classic genetic diseases everyone learns about in freshman biology.As someone who works in the area of gene therapy everyday,the realistic prospect of curing sickle cell disease with gene therapy is simply astonishing,''
''This provides hope that the next generation of students will read about the devastation of this disease in history books only.''
Dr.Gary designed the viral vector for the giant axonal neuropathy gene therapy clinical trial and others.The experimental gene therapy took place at the Necker Children's hospital in Paris.
Dr.Philippe Leboulch an author of the research and Professor of Medicine said:
''Since therapy was applied,he hasn't had any pain,any complications,he plays sports and goes to school;so we are quite pleased with the results.''
Dr.Trish Wong of Oregon Health and Science university says,the new study is truly amazing work,proof of principle that a cure for this chronic,devastating disease is in sight.
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