Genome-Edited Adult Stem Cells: Next-Generation Advanced Therapy Medicinal Products

Dear Colleagues,

The development of advanced therapy medicinal products (ATMPs) has progressed significantly in recent years, especially with the FDA/EMA approval of chimeric antigen receptor T cells (CAR-T) field. To date, those ‘living drugs’ are based mainly on engineered cells with viral vectors, but genome editing (GE) of adult cells is now offering groundbreaking opportunities for the next generation of ATPMs. Recent improvements of GE technologies have unlocked the option of directly targeting and modifying genomic sequences for clinical application in a more precise manner in the field of cancer immunotherapy, HIV infection, and hematological and hereditary diseases. As result, there is an Increasing number of clinical trials using zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), clustered regularly interspaced short palindromic repeat (CRISPR)–Cas-associated nucleases, and base editing (BE) tools for repurposing the functionality (knock-out, knock-in) of a different range of adult cells (such as T cells, NK cells, or hematopoietic progenitor cells). However, beyond the many benefits of GE-directed therapy, there are some challenges that should be optimized in terms of clinical manufacturing, risk of toxicities, and selection of target genes to generate a more homogeneous and robust ATMP.

This Special Issue will collect selected contributions of GE-ATMPs oriented toward clinical development, manufacturing, optimization of GE tools for clinical application, innovative approaches for the generation of novel genome-edited adult cells, and evaluation of quality, safety, and regulatory aspects of GE-ATPMs. We are pleased to invite review articles or original research papers which address the above mentioned topics and look forward to reading your contributions.

Dr. María Tristán-Manzano
Dr. Francisco Martín
Guest Editors

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• genome editing of advanced medicinal products (ATMP)
• CRISPRCas
• TALENs
• ZFN
• base editors
• large deletions
• off-targets
• safety profile
• regulatory affairs of edited cells
• edited CAR-T cells
• manufacturing of genome-edited ATMPs