@article{overton_boi_shastry_smith-moore_balchunas_sambandan_gilleskie_2023, title={Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy}, volume={34}, ISSN={1043-0342 1557-7422}, url={http://dx.doi.org/10.1089/hum.2022.235}, DOI={10.1089/hum.2022.235}, abstractNote={The manufacturing of gene therapy products is a rapidly growing industry bolstered by the tremendous potential of these therapies to provide lifesaving treatment for rare and complex genetic diseases. The industry's steep rise has resulted in a high demand for skilled staff required to manufacture gene therapy products of the expected high quality. To address this skill shortage, more opportunities for education and training in all aspects of gene therapy manufacturing are needed. The Biomanufacturing Training and Education Center (BTEC) at the North Carolina State University (NC State) has developed and delivered (and continues to deliver) a four-day, hands-on course titled Hands-On cGMP Biomanufacturing of Vectors for Gene Therapy. The course, which consists of 60% hands-on laboratory activities and 40% lectures, aims to provide a comprehensive understanding of the gene therapy production process, from vial thaw through the final formulation step, and analytical testing. This paper discusses the design of the course, the backgrounds of the nearly 80 students who have participated in the seven offerings held since March 2019, and feedback from the course participants.}, number={7-8}, journal={Human Gene Therapy}, publisher={Mary Ann Liebert Inc}, author={Overton, Laurie and Boi, Cristiana and Shastry, Shriarjun and Smith-Moore, Caroline and Balchunas, John and Sambandan, Deepa and Gilleskie, Gary}, year={2023}, month={Apr}, pages={259–272} }
 @article{wang_cho_campbell_panduri_coviello_caballero_sambandan_kleeberger_polack_ofman_et al._2022, title={Epigenome-wide association study of bronchopulmonary dysplasia in preterm infants: results from the discovery-BPD program}, volume={14}, ISSN={["1868-7083"]}, DOI={10.1186/s13148-022-01272-0}, abstractNote={AbstractBackgroundBronchopulmonary dysplasia (BPD) is a lung disease in premature infants caused by therapeutic oxygen supplemental and characterized by impaired pulmonary development which persists into later life. While advances in neonatal care have improved survival rates of premature infants, cases of BPD have been increasing with limited therapeutic options for prevention and treatment. This study was designed to explore the relationship between gestational age (GA), birth weight, and estimated blood cell-type composition in premature infants and to elucidate early epigenetic biomarkers associated with BPD.MethodsCord blood DNA from preterm neonates that went on to develop BPD (n = 14) or not (non-BPD,n = 93) was applied to Illumina 450 K methylation arrays. Blood cell-type compositions were estimated using DNA methylation profiles. Multivariable robust regression analysis elucidated CpGs associated with BPD risk. cDNA microarray analysis of cord blood RNA identified differentially expressed genes in neonates who later developed BPD.ResultsThe development of BPD and the need for oxygen supplementation were strongly associated with GA (BPD,p < 1.0E−04; O2supplementation,p < 1.0E−09) and birth weight (BPD,p < 1.0E−02; O2supplementation,p < 1.0E−07). The estimated nucleated red blood cell (NRBC) percent was negatively associated with birth weight and GA, positively associated with hypomethylation of the tobacco smoke exposure biomarker cg05575921, and high-NRBC blood samples displayed a hypomethylation profile. Epigenome-wide association study (EWAS) identified 38 (Bonferroni) and 275 (false discovery rate 1%) differentially methylated CpGs associated with BPD. BPD-associated CpGs in cord blood were enriched for lung maturation and hematopoiesis pathways. Stochastic epigenetic mutation burden at birth was significantly elevated among those who developed BPD (adjustedp = 0.02). Transcriptome changes in cord blood cells reflected cell cycle, development, and pulmonary disorder events in BPD.ConclusionsWhile results must be interpreted with caution because of the small size of this study, NRBC content strongly impacted DNA methylation profiles in preterm cord blood and EWAS analysis revealed potential insights into biological pathways involved in BPD pathogenesis.Graphical Abstract}, number={1}, journal={CLINICAL EPIGENETICS}, author={Wang, Xuting and Cho, Hye-Youn and Campbell, Michelle R. and Panduri, Vijayalakshmi and Coviello, Silvina and Caballero, Mauricio T. and Sambandan, Deepa and Kleeberger, Steven R. and Polack, Fernando P. and Ofman, Gaston and et al.}, year={2022}, month={Dec} }