Results 21 to 30 of about 51,551 (285)

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

Effects of Immunization with Recombinant Schistosoma mansoni Enzymes AK and HGPRT: Murine Infection Control

Pathogens, 2023
Schistosomiasis is one of the most important human helminthiases worldwide. Praziquantel is the current treatment, and no vaccine is available until the present.
Ana Carolina Maragno Fattori, Elisandra de A. Montija, Bruna D. de L. Fragelli, Ricardo de O. Correia, Cynthia Aparecida de Castro, Larissa Romanello, Camila T. Nogueira, Silmara M. Allegretti, Edson G. Soares, Humberto D. Pereira, Fernanda de F. Anibal   +10 more
doaj   +1 more source

Structure, expression, and mutation of the hypoxanthine phosphoribosyltransferase gene.

Proceedings of the National Academy of Sciences of the United States of America, 1984
D. Melton, D. Konecki, J. Brennand, C. Caskey   +3 more
semanticscholar   +3 more sources

Research Advances in Purine Phosphoribosyltransferases of Protozoan Parasites

Guangdong nongye kexue, 2023
Parasitic protozoa are single-celled organisms that have adapted to live in cells of humans and animals. The protozoan parasites include Leishmania spp., Trypanosoma spp., Plasmodium spp., Toxoplasma gondii, Cryptosporidium spp., and Eimeria spp., which ...
Zhihui YU, Siyun FANG, Mingfei SUN, Nanshan QI, Wenjun LIU, Juan LI, Junjing HU, Shenquan LIAO   +7 more
doaj   +1 more source

Analysis of parameters of purine metabolism in patients with diabetes mellitus type 2

Medičnì Perspektivi, 2023
The aim of this research is to comprehensively analyze the current state of purine metabolism as well as assess its features in patients with diabetes mellitus type 2, taking into account the clinical and metabolic polymorphism of the disease.
A.O. Chernyaeva, M.R. Mykytyuk , Yu.I. Karachentsev   +2 more
doaj   +1 more source

Targeted Knock-in of Transgenes into the CHO Cell Genome Using CRISPR-mediated Integration Systems [PDF]

MATEC Web of Conferences, 2021
Biopharmaceutical proteins are usually produced by culturing recombinant Chinese hamster ovary (CHO) cells. High producer cell lines are screened from transfected cells with random integration of target genes. Since transgene expression is susceptible to
Iwao Ryusei, Kawabe Yoshinori, Murakami Mai, Ito Akira, Kamihira Masamichi   +4 more
doaj   +1 more source

PRTFDC1 is a genetic modifier of HPRT-deficiency in the mouse. [PDF]

PLoS ONE, 2011
Lesch-Nyhan disease (LND) is a severe X-linked neurological disorder caused by a deficiency of hypoxanthine phosphoribosyltransferase (HPRT). In contrast, HPRT-deficiency in the mouse does not result in the profound phenotypes such as self-injurious ...
Alaine C Keebaugh, Heather A Mitchell, Meriem Gaval-Cruz, Kimberly G Freeman, Gaylen L Edwards, David Weinshenker, James W Thomas   +6 more
doaj   +1 more source

Normal Uricemia in Lesch–Nyhan Syndrome and the Association with Pulmonary Embolism in a Young Child—A Case Report and Literature Review

Pediatrics and Neonatology, 2014
Deficiency of hypoxanthine phosphoribosyltransferase activity is a rare inborn error of purine metabolism with subsequent uric acid overproduction and neurologic presentations.
Jeng-Dau Tsai, Shan-Ming Chen, Chien-Heng Lin, Min-Sho Ku, Teng-Fu Tsao, Ji-Nan Sheu   +5 more
doaj   +1 more source

Chinese Hamster Hypoxanthine-Guanine Phosphoribosyltransferase

Journal of Biological Chemistry, 1974
Anne S. Olsen, Gregory Milman
openalex   +2 more sources

Reduced levels of dopamine and altered metabolism in brains of HPRT knock-out rats: a new rodent model of Lesch-Nyhan Disease [PDF]

, 2016
Lesch-Nyhan disease (LND) is a severe neurological disorder caused by loss-of-function mutations in the gene encoding hypoxanthine phosphoribosyltransferase (HPRT), an enzyme required for efficient recycling of purine nucleotides.
AM Geurts, C Tong, CL Wu, D Pelled, DJ Creek, DJ Williamson, E Zoref-Shani, GD Zheng, GH Guibinga, GH Guibinga, GR Breese, HA Jinnah, HA Jinnah, HA Jinnah, HA Simmonds, K Blair, KG Lloyd, M Buehr, M Hooper, M Lesch, M Mazzali, MA Cenci, MG Garcia, MK Dienhart, MR Kuehn, P Li, QL Ying, RJ Torres, S Brosh, S Finger, S Meek, S Meek, S Wolf, SB Dunnett, SL Meddle, SM Downs, SM Downs, TJ Aitman, TP Yang, TP Zwaka, WJ Tax, WL Nyhan, WL Nyhan, X Zheng, Y Chen, Y Yu   +45 more
core   +4 more sources