Results 191 to 200 of about 95,867 (295)
Biotechnology and Applied Biochemistry, EarlyView.ABSTRACT Rolling‐circle amplification (RCA) is a completely synthetic and rapid mechanism for scaling‐up bulk DNA that eliminates requirements for large‐scale bacterial fermentation. Several reports have described the surprising ability of large unprocessed (hyperbranched) rolling circle‐amplified DNA (RCA DNA) to effectively transfect cultured cells ...
Evelina Loghin +8 more
wiley +1 more source
CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors. [PDF]
Cell Rep, 2017 Bak RO, Porteus MH.
europepmc +1 more source
Episomal Expression Cassettes For Gene Therapy
, 2007 The Invention Consists Of Episomal Expression Cassettes For Expression Of A Transgene In Gene Therapy. The Expression Cassettes Consist Of Regulatory Elements Of The Human Cytokeratin Gene And A Transgene. The Invention Also Includes Of Liposomes For Transfection Of Epithelial Tissue With The Cassettes In Treatment Of Cystic Fibrosis, Emphysema ...
openaire +1 more source
British Journal of Clinical Pharmacology, EarlyView.While the event‐free survival (EFS) of children treated for acute lymphoblastic leukaemia (ALL) has improved greatly in the last decades, the EFS for patients diagnosed with ALL before the age of one is still under 50%. This outcome further decreases when infants have a rearrangement in the gene encoding histone‐lysine N‐methyltransferase 2A (KMT2A ...
Tirsa de Kluis +5 more
wiley +1 more source
Efficacy, safety and cost‐effectiveness of CAR‐T therapy
British Journal of Clinical Pharmacology, EarlyView.CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher +7 more
wiley +1 more source
CAQ Corner: Basic concepts of transplant immunology
, 2022 Liver Transplantation, EarlyView.
Amanda Cheung, Josh Levitsky
wiley +1 more source
Tetracycline, sulfonamide and trimethoprim resistance genes and integrons in Escherichia coli [PDF]
, 2014 Argudín, MA +7 more
core +1 more source
British Journal of Clinical Pharmacology, EarlyView.Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić +7 more
wiley +1 more source
Versatile nourseothricin and streptomycin/spectinomycin resistance gene cassettes and their use in chromosome integration vectors. [PDF]
J Microbiol Methods, 2016 Lehman SS +5 more
europepmc +1 more source
Biotechnology and Bioengineering, EarlyView.ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai +9 more
wiley +1 more source