Breaking News
June 24, 2018 - Enlist a Pharmacist to Help Manage High Blood Pressure
June 24, 2018 - Genes found related to the reduction of proteins that contribute to Alzheimer’s onset
June 24, 2018 - 1 in 5 immigrant children detained during ‘zero tolerance’ border policy are under 13
June 24, 2018 - Personal automated cell lab assistant from Leica saves time with quality results
June 24, 2018 - Drug use can have social benefits, and acknowledging this could improve rehabilitation
June 24, 2018 - AMSBIO introduces MyEZGel 3D-iPSC Matrix for more accurate in vivo predictions
June 24, 2018 - RaySearch releases new RayStation 8A to expand support for TomoTherapy platform
June 24, 2018 - Dying cancer cells make remaining glioblastoma cells more aggressive and therapy-resistant
June 24, 2018 - Researchers discover new type of cell that hinders formation of fat cells
June 24, 2018 - Scientists develop unique program to predict a form of Parkinson’s disease
June 24, 2018 - Adult Obesity Prevalence Varies With Level of Urbanization
June 24, 2018 - Picking an exercise boot camp
June 24, 2018 - Researchers outline a connection between subplate neurons and brain disorders
June 24, 2018 - Four cups of coffee a day shown to protect heart muscle
June 24, 2018 - ‘Antifreeze’ molecules may hold key to better treatments for brain injuries
June 24, 2018 - Opening onsite health clinics for workers can cut health care costs
June 24, 2018 - Glooko to demonstrate new version of diabetes management mobile application at ADA meeting
June 24, 2018 - Florida Teen First Human Case of Another Mosquito-Borne Virus
June 24, 2018 - Blood type O patients may have higher risk of death from severe trauma
June 24, 2018 - New studies on molecular and cellular proteomics
June 24, 2018 - Algorithm predicts dangerous low blood pressure during surgery
June 24, 2018 - Herpes may play role in pathogenesis of Alzheimer’s
June 24, 2018 - Inaccurate measurement of sodium intake may account for paradoxical results, study suggests
June 24, 2018 - Aquinnah Pharmaceuticals wins NINDS grant to advance novel therapies for ALS
June 24, 2018 - Study upends conventional view of opioid mechanism of action
June 24, 2018 - Floppy eyelids may be sign of sleep apnea, study finds
June 23, 2018 - Researchers highlight new nurse training model to address shortage of primary care
June 23, 2018 - New Olympus cellSens 2.1 speeds up image analysis
June 23, 2018 - Attitudes Among Obese Are Not Aligned With Healthy Living
June 23, 2018 - Early birds less prone to depression
June 23, 2018 - Scientists use novel approach to uncover how brain networks interact to make word-choice decisions
June 23, 2018 - Researchers discover shared genetic basis for psychiatric disorders
June 23, 2018 - Study shows fat cells increase in size and number upon exposure to fracking chemicals
June 23, 2018 - Water-limited landscapes can facilitate disease transmission
June 23, 2018 - Exercise May Ease Inflammation Tied to Obesity
June 23, 2018 - Is it their own fault?! How people judge the exclusion of others
June 23, 2018 - Researchers use advanced technology to identify proteomes of Th17 and iTreg cells
June 23, 2018 - Researchers develop low-cost plastic sensors to monitor wide range of health conditions
June 23, 2018 - Lipid-scrambling DNA enzyme outperforms naturally occurring counterpart, say researchers
June 23, 2018 - Apps for children should emphasize parent and child choice, researchers say
June 23, 2018 - Teenage girls report higher degree of daytime sleepiness than boys
June 23, 2018 - Protein Data Bank at Rutgers impacts research, education and drug discovery
June 23, 2018 - Study unravels new piece of information in the Huntington’s disease puzzle
June 23, 2018 - Scientists develop new device to test cancer drug combinations quickly and cheaply
June 23, 2018 - Neural Analytics wins CE Mark for NeuralBot System
June 23, 2018 - Infant omega-3 supplementation tied to decreased waist size
June 23, 2018 - Massive analysis of genomes reveals insights into genetic overlap among psychiatric diseases
June 23, 2018 - New therapeutic approach may delay neurodegeneration in rare genetic disease
June 23, 2018 - Broken shuttle protein may hinder learning in patients with brain disorders
June 23, 2018 - Study finds increase in daily cannabis use among American adults
June 23, 2018 - Researchers create electronic skin that brings back real sense of touch to prosthetic limbs
June 23, 2018 - FIRS: Guidance Offered for Protecting Youth From E-Cigarettes
June 23, 2018 - Scientists unravel molecular mechanisms of Parkinson’s disease
June 23, 2018 - When the Heart Stops, Drugs Often to Blame
June 23, 2018 - Scientists show that a key Parkinson’s biomarker can be identified in the retina
June 23, 2018 - Study finds factors underlying current rise in radicalization among European youth
June 23, 2018 - New study finds higher heart disease risk in bisexual men
June 23, 2018 - Coconut oil diet increases vitality, lifespan of fruit flies with peroxisomal disorder
June 23, 2018 - Jumping genes or transposons and their role in the genetic code
June 23, 2018 - The Current issue of “The view from here” is concerned with Therapeutics
June 23, 2018 - Abnormal lipid metabolism in fat cells predicts future weight gain and diabetes in women
June 23, 2018 - Alcohol problems linked to sex without condom use among black gay men
June 23, 2018 - DNA patterns in circulating blood cells can help identify spastic cerebral palsy
June 23, 2018 - Unsubstantiated health claims widespread within weight loss industry
June 23, 2018 - FDA grants marketing authorization for use of two catheter-based devices in hemodialysis patients
June 23, 2018 - An ingrown toenail not the same as a bypass
June 23, 2018 - Study suggests proteinuria lowering as important target in managing pediatric CKD
June 23, 2018 - Dynamic model helps make predictions about gut microbiome
June 23, 2018 - Research consortium wins £2.9 million to help tackle antibacterial resistance in Thailand
June 23, 2018 - Schizophrenia patients account for over 1 in 10 suicide deaths, study shows
June 23, 2018 - Overdose risk increases five-fold with concurrent opioid and benzodiazepine use
June 23, 2018 - FDA Alert: Kratom (mitragyna speciosa) powder products by Gaia Ethnobotanical: Recall
June 23, 2018 - Study highlights inadequate effort of health care insurers to combat opioid epidemic
June 23, 2018 - CDC chief asks for, and gets, cut to his record $375K pay
June 22, 2018 - Novel cellular pathway may clarify how arterial inflammation develops into atherosclerosis
June 22, 2018 - Pioneering exercise program improves physical, mental health of elderly people living in care homes
June 22, 2018 - Rutgers Cancer Institute educates childhood cancer survivors about late effects of treatment
June 22, 2018 - Study tests accuracy of device designed to detect heart dysfunction in childhood cancer survivors
June 22, 2018 - Study links annual haze with increased hospitalizations for respiratory problems
June 22, 2018 - Robotic surgery appears to be as effective as open surgery in treating bladder cancer
Gene Therapy Found to Be Promising for β-Thalassemia

Gene Therapy Found to Be Promising for β-Thalassemia

image_pdfDownload PDFimage_print

THURSDAY, April 19, 2018 — For patients with severe β-thalassemia, the need for red-cell transfusions can be reduced or eliminated by transducing cells with the LentiGlobin BB305 vector, according to a study published in the April 18 issue of the New England Journal of Medicine.

Alexis A. Thompson, M.D., M.P.H., from the Northwestern University Feinberg School of Medicine in Chicago, and colleagues obtained mobilized autologous CD34+ cells from 22 patients with transfusion-dependent β-thalassemia and transduced the cells with LentiGlobin BB305 vector, which encodes adult hemoglobin with a T87Q amino acid substitution. After patients underwent myeloablative busulfan conditioning, the cells were reinfused and patients were monitored for adverse events, vector integration, and levels of replication-competent lentivirus.

The researchers found that all but one of the 13 patients with a non-β00 genotype had stopped receiving red-cell transfusions at a median of 26 months after infusion of the gene-modified cells. In evaluated patients with hemoglobin levels near normal ranges, correction of biologic markers of dyserythropoiesis was achieved. The median annualized transfusion volume was decreased by 73 percent in nine patients with a β00 genotype or two copies of the IVS1-110 mutation. In three patients, red-cell transfusions were discontinued. Adverse events related to treatment were typical of those associated with autologous stem-cell transplantation.

“Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long-term red-cell transfusions in 22 patients with severe β-thalassemia without serious adverse events related to the drug product,” the authors write.

The study was funded by Bluebird Bio.

Abstract/Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)

© 2018 HealthDay. All rights reserved.

Posted: April 2018

Tagged with:

About author

Related Articles