Long-term risks of gene therapy are not fully understood. In this study, we evaluated safety outcomes in 783 patients over more than 2,200 total patient-years of observation from 38 T cell therapy trials. The trials employed integrating gammaretroviral or lentiviral vectors to deliver engineered receptors to target HIV-1 infection or cancer.
View Article and Find Full Text PDFUnlabelled: The past decade has seen remarkable progress in identifying genes that, when impacted by deleterious coding variation, confer high risk for autism spectrum disorder (ASD), intellectual disability, and other developmental disorders. However, most underlying gene discovery efforts have focused on individuals of European ancestry, limiting insights into genetic risks across diverse populations. To help address this, the Genomics of Autism in Latin American Ancestries Consortium (GALA) was formed, presenting here the largest sequencing study of ASD in Latin American individuals (n>15,000).
View Article and Find Full Text PDFBackground: HSCT conditioning regimens cause massive lysis of hematopoietic cells with release of toxic intracellular molecules into the circulation.
Objectives: To describe the response to oxidative stress early after hemopoietic stem cell transplantation (HSCT) and assess the association of early oxidative stress with later transplant outcomes.
Study Design: Key components of in the body's physiological response to oxidative stress were studied in a cohort of 122 consecutive pediatric allogeneic HSCT recipients.
Dimension-engineered synthesis of atomically thin II-VI nanoplatelets (NPLs) remains an open challenge. While CdSe NPLs have been made with confinement ranging from 2 to 11 monolayers (ML), CdTe NPLs have been significantly more challenging to synthesize and separate. Here we provide detailed mechanistic insight into the layer-by-layer growth kinetics of the CdTe NPLs.
View Article and Find Full Text PDFObjectives: Given the rapid growth of the field of health policy and systems research (HPSR), it is important to monitor the research environment, especially the evolution of HPSR research outputs in low- and middle-income countries (LMICs). The objective of this study was to generate quantitative metrics to assess the production of HPSR publications and the role of the Alliance for Health Policy and Systems Research (the Alliance) grant-funded projects in 11 LMICs over the past 20 years.
Methods: We conducted a systematic literature search for HPSR literature from 1999 to 2020 pertaining to 11 target LMIC countries, including grey literature.