Enrichment strategies to enhance genome editing

NS Mikkelsen, RO Bak - Journal of Biomedical Science, 2023 - Springer
Genome editing technologies hold great promise for numerous applications including the
understanding of cellular and disease mechanisms and the development of gene and …

Potency assays and biomarkers for cell-based advanced therapy medicinal products

C Capelli, C Cuofano, C Pavoni, S Frigerio… - Frontiers in …, 2023 - frontiersin.org
Advanced Therapy Medicinal Products (ATMPs) based on somatic cells expanded in vitro,
with or without genetic modification, is a rapidly growing area of drug development, even …

Combining different CRISPR nucleases for simultaneous knock-in and base editing prevents translocations in multiplex-edited CAR T cells

V Glaser, C Flugel, J Kath, W Du, V Drosdek, C Franke… - Genome Biology, 2023 - Springer
Background Multiple genetic modifications may be required to develop potent off-the-shelf
chimeric antigen receptor (CAR) T cell therapies. Conventional CRISPR-Cas nucleases …

Precise CRISPR-Cas9 gene repair in autologous memory T cells to treat familial hemophagocytic lymphohistiocytosis

X Li, T Wirtz, T Weber, M Lebedin, ED Lowenstein… - Science …, 2024 - science.org
Familial hemophagocytic lymphohistiocytosis (FHL) is an inherited, often fatal immune
deficiency characterized by severe systemic hyperinflammation. Although allogeneic bone …

Delivery of macromolecules in unstimulated T cells by photoporation with polydopamine nanoparticles

D Berdecka, A Harizaj, I Goemaere, D Punj… - Journal of Controlled …, 2023 - Elsevier
Ex vivo modification of T cells with exogenous cargo is a common prerequisite for the
development of T cell therapies, such as chimeric antigen receptor therapy. Despite the …

GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy

M Caforio, S Iacovelli, C Quintarelli, F Locatelli… - Journal of Experimental …, 2024 - Springer
Background CRISPR/Cas9 system to treat human-related diseases has achieved significant
results and, even if its potential application in cancer research is improving, the application …

Engineering challenges and opportunities in autologous cellular cancer immunotherapy

CR Foley, SL Swan, MA Swartz - The Journal of Immunology, 2024 - journals.aai.org
The use of a patient's own immune or tumor cells, manipulated ex vivo, enables Ag-or
patient-specific immunotherapy. Despite some clinical successes, there remain significant …

CRISPR-Cas and CRISPR-based screening system for precise gene editing and targeted cancer therapy

M Qin, C Deng, L Wen, G Luo, Y Meng - Journal of Translational Medicine, 2024 - Springer
Target cancer therapy has been developed for clinical cancer treatment based on the
discovery of CRISPR (clustered regularly interspaced short palindromic repeat)-Cas system …

Integration of ζ-deficient CARs into the CD3ζ gene conveys potent cytotoxicity in T and NK cells

J Kath, C Franke, V Drosdek, W Du, V Glaser… - Blood, 2024 - ashpublications.org
Chimeric antigen receptor (CAR)-redirected immune cells hold significant therapeutic
potential for oncology, autoimmune diseases, transplant medicine, and infections. All …

Graphene-encapsulated iron nanoparticles as a non-viral vector for gene delivery into melanoma cells

M Bamburowicz-Klimkowska, M Malecki… - Biochemical and …, 2023 - Elsevier
The rapid progress of nanotechnology has led to use different nanomaterials for biomedical
applications. Among them, graphene-encapsulated magnetic nanoparticles (GEMNS) are …