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2012 2
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Page 1
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
After previously establishing that lentiviral transfer of a marked beta-globin (beta(A-T87Q)) gene could substitute for long-term red-cell transfusions in a patient with beta-thalassemia, we wanted to evaluate the safety and efficacy of such gene therapy in patients with t …
After previously establishing that lentiviral transfer of a marked beta-globin (beta(A-T87Q)) gene could substitute for long-term red …
Long-Term Complications in Youth-Onset Type 2 Diabetes.
TODAY Study Group; Bjornstad P, Drews KL, Caprio S, Gubitosi-Klug R, Nathan DM, Tesfaldet B, Tryggestad J, White NH, Zeitler P. TODAY Study Group, et al. N Engl J Med. 2021 Jul 29;385(5):416-426. doi: 10.1056/NEJMoa2100165. N Engl J Med. 2021. PMID: 34320286 Free PMC article.
Long-term Outcomes With Islet-Alone and Islet-After-Kidney Transplantation for Type 1 Diabetes in the Clinical Islet Transplantation Consortium: The CIT-08 Study.
Rickels MR, Eggerman TL, Bayman L, Qidwai JC, Alejandro R, Bridges ND, Hering BJ, Markmann JF, Senior PA, Hunsicker LG; Clinical Islet Transplantation Consortium. Rickels MR, et al. Diabetes Care. 2022 Oct 17;45(12):2967-75. doi: 10.2337/dc21-2688. Online ahead of print. Diabetes Care. 2022. PMID: 36250905 Free PMC article.
OBJECTIVE: To determine long-term outcomes for islet-alone and islet-after-kidney transplantation in adults with type 1 diabetes complicated by impaired awareness of hypoglycemia. ...Insulin independence was achieved by 74% of islet-alone and islet-after-kidney transplanta …
OBJECTIVE: To determine long-term outcomes for islet-alone and islet-after-kidney transplantation in adults with type 1 diabetes comp …
Endocrine-sensitive physical endpoints in newborns: ranges and predictors.
Shah R, Alshaikh B, Schall JI, Kelly A, Ford E, Zemel BS, Umbach DM, Adgent M, Stallings VA. Shah R, et al. Pediatr Res. 2021 Feb;89(3):660-666. doi: 10.1038/s41390-020-0950-2. Epub 2020 May 12. Pediatr Res. 2021. PMID: 32396926 Free PMC article.
Studies using standardized measurement techniques that describe demographic and clinical variability in these endpoints are lacking. METHODS: Three hundred and eighty-eight healthy term newborns <3 days old were evaluated, 69% African American and 25% White. ...
Studies using standardized measurement techniques that describe demographic and clinical variability in these endpoints are lacking. METHODS …
Impact of Genotype, Serum Bile Acids, and Surgical Biliary Diversion on Native Liver Survival in FIC1 Deficiency.
van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipiński P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Muñoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsőfi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz Ö, Kerkar N, Hørby Jørgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul WW, Hansen B, Verkade HJ; Natural Course and Prognosis of PFIC and Effect of Biliary Diversion Consortium. van Wessel DBE, et al. Hepatology. 2021 Aug;74(2):892-906. doi: 10.1002/hep.31787. Epub 2021 Jul 13. Hepatology. 2021. PMID: 33666275 Free PMC article.
The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating mutations (PPTMs), and possible associations of serum bile acid (sBA) concentrations and surgical biliary diversion (SBD) with long-term …
The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating muta …
Engineered Human Gastrointestinal Cultures to Study the Microbiome and Infectious Diseases.
Blutt SE, Crawford SE, Ramani S, Zou WY, Estes MK. Blutt SE, et al. Cell Mol Gastroenterol Hepatol. 2017 Dec 9;5(3):241-251. doi: 10.1016/j.jcmgh.2017.12.001. eCollection 2018 Mar. Cell Mol Gastroenterol Hepatol. 2017. PMID: 29675450 Free PMC article. Review.
The continued development of these cultures to incorporate more facets of the gastrointestinal tract, including neurons, immune cells, and the microbiome, will unravel new mechanisms regulating host-microbial interactions with the long-term goal of translating findings int …
The continued development of these cultures to incorporate more facets of the gastrointestinal tract, including neurons, immune cells, and t …
Pulmonary Vasodilator Therapy in Shock-associated Cardiac Arrest.
Morgan RW, Sutton RM, Karlsson M, Lautz AJ, Mavroudis CD, Landis WP, Lin Y, Jeong S, Craig N, Nadkarni VM, Kilbaugh TJ, Berg RA. Morgan RW, et al. Am J Respir Crit Care Med. 2018 Apr 1;197(7):905-912. doi: 10.1164/rccm.201709-1818OC. Am J Respir Crit Care Med. 2018. PMID: 29244522 Free PMC article.
OBJECTIVES: To determine if the addition of inhaled nitric oxide (iNO) to hemodynamic-directed cardiopulmonary resuscitation (HD-CPR) would improve short-term survival from cardiac arrest associated with shock and systemic inflammation. ...CONCLUSIONS: The addition of iNO …
OBJECTIVES: To determine if the addition of inhaled nitric oxide (iNO) to hemodynamic-directed cardiopulmonary resuscitation (HD-CPR) would …
Evaluation of a large-scale aptamer proteomics platform among patients with kidney failure on dialysis.
Ren Y, Ruan P, Segal M, Dobre M, Schelling JR, Banerjee U, Shafi T, Ganz P, Dubin RF; CRIC Study Investigators. Ren Y, et al. PLoS One. 2023 Dec 11;18(12):e0293945. doi: 10.1371/journal.pone.0293945. eCollection 2023. PLoS One. 2023. PMID: 38079395 Free PMC article.
Long-term (one year) variability was studied in 421 participants in the Chronic Renal Insufficiency Cohort. ...RESULTS: In ANML format, median[IQR] intra-assay coefficient of variation (CV) was 2.38%[1.76, 3.40] and inter-assay CV was 7.38%[4.61, 13.12]. Short-term …
Long-term (one year) variability was studied in 421 participants in the Chronic Renal Insufficiency Cohort. ...RESULTS: In ANML forma …
Total Serum Cholesterol and Pancreatic Cancer: A Nested Case-Control Study.
Chen WC, Boursi B, Mamtani R, Yang YX. Chen WC, et al. Cancer Epidemiol Biomarkers Prev. 2019 Feb;28(2):363-369. doi: 10.1158/1055-9965.EPI-18-0421. Epub 2018 Oct 17. Cancer Epidemiol Biomarkers Prev. 2019. PMID: 30333217
No significant association was seen at subsequent discrete intervals before index date. CONCLUSIONS: TSC is a significant predictor of short-term risk for pancreatic cancer. This risk increase associated with lower TSC was independent of statins. ...
No significant association was seen at subsequent discrete intervals before index date. CONCLUSIONS: TSC is a significant predictor of short …
65 results