Results 51 to 60 of about 15,070 (233)

Modeling viral infectious diseases and development of antiviral therapies using human induced pluripotent stem cell-derived systems [PDF]

, 2015
The recent biotechnology breakthrough of cell reprogramming and generation of induced pluripotent stem cells (iPSCs), which has revolutionized the approaches to study the mechanisms of human diseases and to test new drugs, can be exploited to generate ...
Barzon, Luisa, Berto, Alessandro, Desole, Giovanna, Pacenti, Monia, Palu', Giorgio, Sinigaglia, Alessandro, Trevisan, Marta   +6 more
core   +2 more sources

Historic landmarks in clinical transplantation: Conclusions from the consensus conference at the University of California, Los Angeles [PDF]

, 2000
The transplantation of organs, cells, and tissues has burgeoned during the last quarter century, with the development of multiple new specialty fields.
Brent, LB, Calne, RY, Dausset, JB, Good, RA, Groth, CG, Murray, JE, Schwartz, RS, Shumway, NE, Starzl, TE, Terasaki, PI, Thomas, ED, Van Rood, JJ   +11 more
core   +1 more source

Orotic Aciduria [PDF]

, 2018
Orotic acid is an intermediate found in the pathway for pyrimidine synthesis. The mitochondrial enzyme dihydroorotate dehydrogenase (DHODH) catalyzes the production of orotic acid by the conversion of the compound dihydroorotate to orotic acid.
Fonteh, Aliah L
core   +1 more source

Gene- and Disease-Based Expansion of the Knowledge on Inborn Errors of Immunity

Frontiers in Immunology, 2019
The recent report of the International Union of Immunological Societies (IUIS) has provided the categorized list of 354 inborn errors of immunity. We performed a systematic analysis of genes and diseases from the IUIS report with the use of the OMIM ...
Lyubov E. Salnikova, Lyubov E. Salnikova, Lyubov E. Salnikova, Ekaterina V. Chernyshova, Lyudmila A. Anastasevich, Sergey S. Larin   +5 more
doaj   +1 more source

Microbiome and Its Dysbiosis in Inborn Errors of Immunity

Pathogens, 2023
Inborn errors of immunity (IEI) can present with infections, autoimmunity, lymphoproliferation, granulomas, and malignancy. IEIs are due to genetic abnormalities that disrupt normal host-immune response or immune regulation.
Madhubala Sharma, Manpreet Dhaliwal, Rahul Tyagi, Taru Goyal, Saniya Sharma, Amit Rawat   +5 more
doaj   +1 more source

Genome editing for inborn errors of metabolism: advancing towards the clinic [PDF]

, 2017
Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative.
Bao, Gang, Lee, Ciaran M, Schneller, Jessica L, Venditti, Charles P   +3 more
core   +1 more source

Pathogenesis based Precision Therapy for Inborn Errors of Immunity

罕见病研究, 2022
Precision medicine aims at using target therapy on specific diseases by studying the pathogenesis and finding biomarkers. Inborn errors of immunity (IEI) are caused by single gene mutations, providing the perfect human models to study immunology.
SHU Zhou, MAO Huawei
doaj   +1 more source

Update on inborn errors of immunity

Journal of Allergy and Clinical Immunology
Ever since the first description of an inherited immunodeficiency in 1952 in a boy with gammaglobulin deficiency, new insights have progressed rapidly in disorders that are now referred to as inborn errors of immunity. In a field where fundamental molecular biology, genetics, immune signaling, and clinical care are tightly intertwined, 2022-24 saw a ...
Hanna IJspeert, Emily S.J. Edwards, Robyn E. O’Hehir, Virgil A.S.H. Dalm, Menno C. van Zelm   +4 more
openaire   +2 more sources

Stem Cell-Based Disease Models for Inborn Errors of Immunity

Cells, 2021
The intrinsic capacity of human hematopoietic stem cells (hHSCs) to reconstitute myeloid and lymphoid lineages combined with their self-renewal capacity hold enormous promises for gene therapy as a viable treatment option for a number of immune-mediated ...
Aline Zbinden, Kirsten Canté-Barrett, Karin Pike-Overzet, Frank J. T. Staal   +3 more
doaj   +1 more source

Advances in gene therapy for inborn errors of immunity

Current Opinion in Allergy & Clinical Immunology, 2023
Purpose of review Provide an overview of the landmark accomplishments and state of the art of gene therapy for inborn errors of immunity (IEI). Recent findings Three decades after the first clinical application of gene therapy for IEI, there is one market authorized product ...
Ott de Bruin, Lisa M., Lankester, Arjan C., Staal, Frank J.T.   +2 more
openaire   +2 more sources