UCLA researcher pioneers gene therapy cure for ‘Bubble Baby’ disease
UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with a life-threatening condition called adenosine deaminase–deficient severe combined immunodeficiency, or ADA-deficient SCID. Often called Bubble Baby disease, the condition can be fatal within the first year of life if left untreated. The groundbreaking treatment was developed by Dr. Donald Kohn, a renowned stem cell researcher and member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research . Kohn’s breakthrough was developed over three decades of research aimed at creating a gene therapy that safely restores the immune systems of children with ADA-deficient SCID using the patients’ own cells with no side effects. To date, 18 children with SCID have been cured of the disease after receiving the therapy in clinical trials at UCLA and the National Institutes of Health. “All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy,” said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the UCLA College. “Instead they are all thriving with fully functioning immune systems.” Children born with SCID are kept in controlled, isolated environments because without an immune system, ordinary illness and infection could be lethal. “Other current options for treating ADA-deficient SCID ...
Conclusion: Polymorphisms in the rs7557939 region of the BCL11A gene appear to somehow interfere in the clinical setting of patients with SCD, suggesting relation with the concentration of MetHb and LDH. This study pioneered an investigation into the association of hemolysis biomarkers with BCL11A gene polymorphisms in SCD.RESUMO Introdu ção: Pacientes com anemia falciforme (AF) apresentam hemólise crônica com biomarcadores séricos aumentados. Os polimorfismos genéticos do gene BLC11A modulam a hemoglobina fetal (HbF), reduzindo, assim, a hemólise. Objetivo: Associar os polim...
We report two cases in which fecal microbiota transplantation used for the treatment of recurrent Clostridium difficile infection were effective in eradicating colonization by carbapenemase-producing Enterobacteriaceae. The presented cases illustrate the potential benefit of fecal microbiota transplantation in resolution of asymptomatic carrier states of multiresistant microorganisms, suggesting the need for further investigations with a view to their applicability in this area.
Officially it ’s a ‘unique holiday hotel for cancer patients and those with life-threatening illnesses’, according to the sign outside the Twenties green-shuttered building in Bournemouth.
Sarah Brookes, 41, a mother of two from Bradford, West Yorkshire, would like commercial sunbeds to be banned outright.
Abstract Sickle cell anemia, a genetic disease caused by a mutation in the beta-globin gene, can present oral manifestations such as delayed tooth eruption and hypomineralized enamel and dentin. The aim of the present study was to evaluate the prevalence and severity of developmental defects of enamel (DDE) and delayed tooth eruption in children with sickle cell anemia. The sample comprised 56 male and female children with sickle cell anemia aged 6 to 12 years and treated at the Hematology and Hemotherapy Center of Pernambuco, Brazil. The data were collected according to the WHO criteria for DDE and tooth eruption. The pre...
ConclusionsIn a setting of short waiting list times, IABP can be used to bridge candidates to urgent HT with acceptable postoperative results, but there were significant rates of adverse clinical events during support.ResumenIntroducción y objetivosEn España, el balón de contrapulsación intraaórtico (BCIA) se ha usado frecuentemente como puente al trasplante cardiaco (TxC) urgente. El propósito es analizar los resultados de esta estrategia.MétodosSe realizó una revisión retrospectiva caso por caso de los registros clínicos de 281 pacientes adultos listad...
Publication date: August 2018Source: Biochimica et Biophysica Acta (BBA) - Reviews on Cancer, Volume 1870, Issue 1Author(s): Chi Van Dang
Publication date: August 2018Source: Biochimica et Biophysica Acta (BBA) - Reviews on Cancer, Volume 1870, Issue 1Author(s):
The steep increase in thyroid cancer diagnosis has not been mirrored by increasing mortality rates and is likely due to widespread overdiagnosis of small neoplasms.
The presence of HER3 expression and a high HER2/CEP17 ratio distinguish a group of invasive HER2-positive ductal breast cancer patients who have a worse prognosis.
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