Results 291 to 300 of about 12,497,887 (407)

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

Nature reviews. Drug discovery, 2019
M. Cavazzana, F. Bushman, A. Miccio, I. André-Schmutz, E. Six   +4 more
semanticscholar   +1 more source

Intein‐based modular chimeric antigen receptor platform for specific CD19/CD20 co‐targeting

Molecular Oncology, EarlyView.
CARtein is a modular CAR platform that uses split inteins to splice antigen‐recognition modules onto a universal signaling backbone, enabling precise, scarless assembly without re‐engineering signaling domains. Deployed here against CD19 and CD20 in B‐cell malignancies, the design supports flexible multi‐antigen targeting to boost T‐cell activation and
Pablo Gonzalez‐Garcia, Noelia Moares, Wenjie Yi‐He, Rosa Luna‐Espejo, Ricardo Fernandez‐Cisnal, Javier Ocaña‐Cuesta, Juan P. Muñoz‐Miranda, Antonio Gabucio, Cecilia M. Fernandez‐Ponce, Francisco Garcia‐Cozar   +9 more
wiley   +1 more source

Microfluidic Platforms for Ex Vivo and In Vivo Gene Therapy. [PDF]

Biosensors (Basel)
Kwak S, Lee H, Yu D, Jeon TJ, Kim SM, Ryu H.   +5 more
europepmc   +1 more source

The rapidly growing landscape of RAS inhibitors: from selective allele blockade to broad inhibition strategies

Molecular Oncology, EarlyView.
RAS‐targeted therapies have become a clinical reality. Although promising results in patients have been achieved, there are still challenges ahead such as coping with drug resistance. Here we summarize selected RAS inhibitors targeting either individual KRAS mutant isoforms (KRASG12C, KRASG12D, KRASG12V) or exhibiting a wider inhibitory spectrum ...
Matthias Drosten, Mariano Barbacid
wiley   +1 more source

P53 Gene Therapy with ZIF-8 Metal-Organic Framework: A Platform in Cancer Gene Therapy. [PDF]

ACS Omega
Salari R, Rastegari B, Hashemi A, Farjadfar A, Masoomi MY.   +4 more
europepmc   +1 more source

A guide to reactive oxygen species in tumour hypoxia: measurement and therapeutic implications

Molecular Oncology, EarlyView.
Hypoxia reshapes tumour redox landscapes by altering compartmental ROS production (mitochondria, NOX, ER, peroxisomes). Accurate interpretation requires oxygen‐contextualised measurement (live biosensors, chemical probes, EPR, LC–MS) and awareness of artefacts (reoxygenation, probe specificity).
Lina Hacker, Elysia Sarsam, Stuart J. Conway, Ester M. Hammond   +3 more
wiley   +1 more source

P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

Genetics in Medicine Open
Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti   +25 more
doaj   +1 more source

Gene therapy ameliorates neuromuscular pathology in CLN3 disease. [PDF]

Acta Neuropathol Commun
Ziółkowska EA, Jablonka-Shariff A, Williams LL, Jansen MJ, Wang SH, Eultgen EM, Wood MD, Hunter DA, Sharma J, Sardiello M, Reese R, Pestronk A, Sands MS, Snyder-Warwick AK, Cooper JD.   +14 more
europepmc   +1 more source

Intra-articular IL-4 gene therapy in arthritis: anti-inflammatory effect and enhanced Th2activity

, 1999
D L Boyle, Kathy Nguyen, Shi‐Mei Zhuang, Yunying Shi, James E. McCormack, Sunil Chada, Gary S. Firestein   +6 more
openalex   +1 more source