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Nature Communications|May 27, 2024
Deciphering bat influenza H18N11 infection dynamics in male Jamaican fruit bats on a single-cell levelSusanne Kessler, Bradly Burke, Geoffroy Andrieux, et al.
Biorxiv : the Preprint Server for Biology|December 20, 2023
Integration of ζ-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cellsJonas Kath, Clemens Franke, Vanessa Drosdek, et al.
Frontiers in Immunology|June 13, 2024
Generating universal anti-CD19 CAR T cells with a defined memory phenotype by CRISPR/Cas9 editing and safety evaluation of the transcriptomeKristina Pavlovic, MDolores Carmona-Luque, Giulia I Corsi, et al.
Blood|March 17, 2024
Integration of ζ-deficient CARs into the CD3ζ gene conveys potent cytotoxicity in T and NK cellsJonas Kath, Clemens Franke, Vanessa Drosdek, et al.
Neurology|January 4, 2013
Pontocerebellar hypoplasia type 1: clinical spectrum and relevance of EXOSC3 mutationsSabine Rudnik-Schöneborn, Jan Senderek, Joanna C Jen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 26, 2021
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaquesAisha A AlJanahi, Cicera R Lazzarotto, Shirley Chen, et al.
Cell Stem Cell|January 28, 2026
Lipid nanoparticle-based non-viral in situ gene editing of congenital ichthyosis-causing mutations in human skin modelsDilem Ceren Apaydin, Gaurav Sadhnani, Tiffany Carlaw, et al.
Journal of Neurology, Neurosurgery, and Psychiatry|December 2, 2023
Recurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesisSafoora B Syeda, Museer A Lone, Payam Mohassel, et al.
Neuromuscular Disorders : NMD|January 6, 2024
270th ENMC International Workshop: Consensus for SMN2 genetic analysis in SMA patients 10-12 March, 2023, Hoofddorp, the NetherlandsEmanuela Abiusi, Mar Costa-Roger, Enrico Silvio Bertini, et al.
Eclinicalmedicine|May 21, 2026
The phenotypic spectrum and genetic determinants of severe spinal muscular atrophy in individuals with a single SMN2 copy: an international retrospective observational studyGianpaolo Cicala, Anna Capasso, Marianna Villa, et al.
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