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2370. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
作者: Salima Hacein-Bey Abina.;H Bobby Gaspar.;Johanna Blondeau.;Laure Caccavelli.;Sabine Charrier.;Karen Buckland.;Capucine Picard.;Emmanuelle Six.;Nourredine Himoudi.;Kimberly Gilmour.;Anne-Marie McNicol.;Havinder Hara.;Jinhua Xu-Bayford.;Christine Rivat.;Fabien Touzot.;Fulvio Mavilio.;Annick Lim.;Jean-Marc Treluyer.;Sébastien Héritier.;Francois Lefrère.;Jeremy Magalon.;Isabelle Pengue-Koyi.;Géraldine Honnet.;Stéphane Blanche.;Eric A Sherman.;Frances Male.;Charles Berry.;Nirav Malani.;Frederic D Bushman.;Alain Fischer.;Adrian J Thrasher.;Anne Galy.;Marina Cavazzana.
来源: JAMA. 2015年313卷15期1550-63页
Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen-matched allogeneic hematopoietic stem cell (HSC) transplantation is often curative but is associated with significant comorbidity.
2371. Effects of high-dose oral insulin on immune responses in children at high risk for type 1 diabetes: the Pre-POINT randomized clinical trial.
作者: Ezio Bonifacio.;Anette-G Ziegler.;Georgeanna Klingensmith.;Edith Schober.;Polly J Bingley.;Marietta Rottenkolber.;Anke Theil.;Anne Eugster.;Ramona Puff.;Claudia Peplow.;Florian Buettner.;Karin Lange.;Jörg Hasford.;Peter Achenbach.; .
来源: JAMA. 2015年313卷15期1541-9页
Exposing the oral mucosa to antigen may stimulate immune tolerance. It is unknown whether treatment with oral insulin can induce a tolerogenic immune response in children genetically susceptible to type 1 diabetes.
2372. Autism occurrence by MMR vaccine status among US children with older siblings with and without autism.
作者: Anjali Jain.;Jaclyn Marshall.;Ami Buikema.;Tim Bancroft.;Jonathan P Kelly.;Craig J Newschaffer.
来源: JAMA. 2015年313卷15期1534-40页
Despite research showing no link between the measles-mumps-rubella (MMR) vaccine and autism spectrum disorders (ASD), beliefs that the vaccine causes autism persist, leading to lower vaccination levels. Parents who already have a child with ASD may be especially wary of vaccinations.
2373. Effect of parent training vs parent education on behavioral problems in children with autism spectrum disorder: a randomized clinical trial.
作者: Karen Bearss.;Cynthia Johnson.;Tristram Smith.;Luc Lecavalier.;Naomi Swiezy.;Michael Aman.;David B McAdam.;Eric Butter.;Charmaine Stillitano.;Noha Minshawi.;Denis G Sukhodolsky.;Daniel W Mruzek.;Kylan Turner.;Tiffany Neal.;Victoria Hallett.;James A Mulick.;Bryson Green.;Benjamin Handen.;Yanhong Deng.;James Dziura.;Lawrence Scahill.
来源: JAMA. 2015年313卷15期1524-33页
Disruptive behavior is common in children with autism spectrum disorder. Behavioral interventions are used to treat disruptive behavior but have not been evaluated in large-scale randomized trials.
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