Results 61 to 70 of about 196,305 (346)

Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Molecular Therapy: Nucleic Acids, 2023
Severe combined immunodeficiency (SCID) is a group of disorders caused by mutations in genes involved in the process of lymphocyte maturation and function.
Ortal Iancu, Daniel Allen, Orli Knop, Yonathan Zehavi, Dor Breier, Adaya Arbiv, Atar Lev, Yu Nee Lee, Katia Beider, Arnon Nagler, Raz Somech, Ayal Hendel   +11 more
doaj   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK

International Journal of Neonatal Screening, 2019
Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds.
Alice Bessey, James Chilcott, Joanna Leaviss, Carmen de la Cruz, Ruth Wong   +4 more
doaj   +1 more source

CD3ε+ Cells in Pigs With Severe Combined Immunodeficiency Due to Defects in ARTEMIS

Frontiers in Immunology, 2020
Severe combined immunodeficiency (SCID) is described as the lack of functional T and B cells. In some cases, mutant genes encoding proteins involved in the process of VDJ recombination retain partial activity and are classified as hypomorphs. Hypomorphic
Adeline N. Boettcher, A. Giselle Cino-Ozuna, Yash Solanki, Jayne E. Wiarda, Jayne E. Wiarda, Jayne E. Wiarda, Ellie Putz, Jeana L. Owens, Sara A. Crane, Amanda P. Ahrens, Crystal L. Loving, Joan. E. Cunnick, Raymond R. R. Rowland, Sara E. Charley, Jack C. M. Dekkers, Christopher K. Tuggle   +15 more
doaj   +1 more source

RNA‐Micelles as Self‐Assembling Structures for Efficient Co‐Delivery of Synergistic siRNA and Nucleoside Analogues to Treat CRC Lung Metastasis

Advanced Functional Materials, EarlyView.
Two kinds of self‐assembled RNA micelles were used to co‐deliver synergistic siRNA and nucleoside analogues for the treatment of colorectal cancer lung metastases. Near‐complete elimination of lung cancer metastases was confirmed in an orthotopic lung metastasis model constructed using human colorectal cancer lung metastases patient surgical samples to
Kai Jin, Piotr Rychahou, Daniel W. Binzel, B. Mark Evers, Peixuan Guo   +4 more
wiley   +1 more source

Treponema denticola in Disseminating Endodontic Infections [PDF]

, 2006
Treponema denticola is a consensus periodontal pathogen that has recently been associated with endodontic pathology. In this study, the effect of mono-infection of the dental pulp with T.
Foschi, F., Izard, Jacques, Müller, R., Prati, C., Sambri, V., Sasaki, H., Stashenko, P.   +6 more
core   +2 more sources

Novel Engraftment and T Cell Differentiation of Human Hematopoietic Cells in ART−/−IL2RG−/Y SCID Pigs

Frontiers in Immunology, 2020
Pigs with severe combined immunodeficiency (SCID) are an emerging biomedical animal model. Swine are anatomically and physiologically more similar to humans than mice, making them an invaluable tool for preclinical regenerative medicine and cancer ...
Adeline N. Boettcher, Yunsheng Li, Amanda P. Ahrens, Matti Kiupel, Kristen A. Byrne, Crystal L. Loving, A. Giselle Cino-Ozuna, Jayne E. Wiarda, Jayne E. Wiarda, Jayne E. Wiarda, Malavika Adur, Blythe Schultz, Jack J. Swanson, Elizabeth M. Snella, Chak-Sum (Sam) Ho, Sara E. Charley, Zoe E. Kiefer, Joan E. Cunnick, Ellie J. Putz, Giuseppe Dell'Anna, Jackie Jens, Swanand Sathe, Frederick Goldman, Erik R. Westin, Jack C. M. Dekkers, Jason W. Ross, Christopher K. Tuggle   +26 more
doaj   +1 more source

Clinical, Immunological, and Molecular Features of Severe Combined Immune Deficiency: A Multi-Institutional Experience From India

Frontiers in Immunology, 2021
BackgroundSevere Combined Immune Deficiency (SCID) is an inherited defect in lymphocyte development and function that results in life-threatening opportunistic infections in early infancy.
Pandiarajan Vignesh, Amit Rawat, Rajni Kumrah, Ankita Singh, Anjani Gummadi, Madhubala Sharma, Anit Kaur, Johnson Nameirakpam, Ankur Jindal, Deepti Suri, Anju Gupta, Alka Khadwal, Biman Saikia, Ranjana Walker Minz, Kaushal Sharma, Mukesh Desai, Prasad Taur, Vijaya Gowri, Ambreen Pandrowala, Aparna Dalvi, Neha Jodhawat, Priyanka Kambli, Manisha Rajan Madkaikar, Sagar Bhattad, Stalin Ramprakash, Raghuram CP, Ananthvikas Jayaram, Meena Sivasankaran, Deenadayalan Munirathnam, Sarath Balaji, Aruna Rajendran, Amita Aggarwal, Komal Singh, Fouzia Na, Biju George, Ankit Mehta, Harsha Prasada Lashkari, Ramya Uppuluri, Revathi Raj, Sandip Bartakke, Kirti Gupta, Sreejesh Sreedharanunni, Yumi Ogura, Tamaki Kato, Kohsuke Imai, Kohsuke Imai, Koon Wing Chan, Daniel Leung, Osamu Ohara, Shigeaki Nonoyama, Michael Hershfield, Yu-Lung Lau, Surjit Singh   +52 more
doaj   +1 more source

Decellularized Extracellular Matrix Scaffolds to Engineer the Dormant Landscape of Microscopic Colorectal Cancer Liver Metastasis

Advanced Healthcare Materials, EarlyView.
Decellularized liver extracellular matrix scaffolds provide a platform to study dormant liver‐metastatic colorectal cancer. They induce reversible dormancy, in combination with nutrient depletion and low dose chemotherapy, through cell cycle arrest and chemotherapy resistance.
Sabrina N. VandenHeuvel, Lucia L. Nash, Abigail J. Clevenger, Claudia A. Collier, Oscar R. Benavides, Sanjana Roy, Brinlee Goggans, Aelita Salikhova, Anvitha Tharakesh, Svasti Haricharan, Amber N. Stratman, Scott Kopetz, Alex J. Walsh, Shreya A. Raghavan   +13 more
wiley   +1 more source

Rescue of Fertility in Homozygous Mice for the Urokinase Plasminogen Activator Transgene by the Transplantation of Mouse Hepatocytes

Cell Transplantation, 2008
Development of the urokinase plasminogen activator/SCID (uPA/SCID) transgenic mouse model has opened new perspectives for the study of different biological mechanisms such as liver regeneration, stem cell differentiation, and human hepatic pathogens.
Nicolas M. Brezillon, Lucie Dasilva, David L'hôte, Florence Bernex, Julie Piquet, Nadine Binart, Serban Morosan, Dina Kremsdorf   +7 more
doaj   +1 more source