Molecular assessment of splicing variants in a cohort of patients with inborn errors of immunity: methodological approach and interpretation remarks by Laura Miguel Berenguel, Carla Gianelli, Carla Gianelli, Carla Gianelli, Elisabet Matas Pérez, Teresa del Rosal, Ana Méndez Echevarría, Ángel Robles Marhuenda, Marta Feito Rodríguez, Maria Teresa Caballero Molina, Maria Teresa Caballero Molina, Lorena Magallares García, Brenda Sánchez Garrido, Samantha Hita Díaz, Luis Allende Martínez, Luis Allende Martínez, Pilar Nozal Aranda, Pilar Nozal Aranda, Pilar Nozal Aranda, Carmen Cámara Hijón, Carmen Cámara Hijón, Carmen Cámara Hijón, Eduardo López Granados, Eduardo López Granados, Eduardo López Granados, Rebeca Rodríguez Pena, Rebeca Rodríguez Pena, Rebeca Rodríguez Pena, María Bravo García-Morato, María Bravo García-Morato, María Bravo García-Morato

“…BackgroundSplicing is the molecular mechanism to produce mature messenger RNA (mRNA) before its translation into protein. …”
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Enhancement of anti‐sarcoma immunity by NK cells engineered with mRNA for expression of a EphA2‐targeted CAR by Pui Yeng Lam, Natacha Omer, Josh K. M. Wong, Cui Tu, Louisa Alim, Gustavo R. Rossi, Maria Victorova, Hannah Tompkins, Cheng‐Yu Lin, Ahmed M. Mehdi, Amos Choo, Melissa R. Elliott, Elaina Coleborn, Jane Sun, Timothy Mercer, Orazio Vittorio, Lachlan J. Dobson, Alexander D. McLellan, Andrew Brooks, Zewen Kelvin Tuong, Seth W. Cheetham, Wayne Nicholls, Fernando Souza‐Fonseca‐Guimaraes

“…Significantly, EphA2‐specific CAR‐NK cells demonstrated enhanced cytotoxic activity against several paediatric sarcoma cell lines in vitro, including those of rhabdomyosarcoma, Ewing sarcoma and osteosarcoma, compared to unmodified NK cells. Transient messenger RNA (mRNA) transfection of NK cells is a safe approach in genetic engineering, with further chemical modifications to mRNA enhancing stability of temporal EphA2‐CAR expression in NK cells, thereby promoting prolonged protein expression. …”
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