Topical Collection "Feature Papers in Gene and Cell Therapy"
Interests: oligonucleotides-based therapeutics; peptide (cell penetrating peptides)-based delivery vectors; antisense oligonucleotides cellular trafficking
Special Issues, Collections and Topics in MDPI journals
Recent progresses in gene and cell therapies have been the incentive for this Biomedicine «Feature Papers in Gene and Cell Therapy» Topical Collection. Contributions from the editorial board members, as well as from distinguished scholars in this rapidly growing field will be acknowledged.
Among the recent achievements in the field of gene therapy, CRISPR-directed gene edition has now reached clinical translation, with promising data in the treatment of sickle-cell anemia and thransthyrethin amyloidosis. The great potential of the technology has lead to partnerships between start-up companies and large pharmaceutical companies. Likewise, the successes of mRNA-based vaccines in the SARS-CoV-2 pandemic is giving rise to many developments of mRNA-based platforms to fight human diseases through vaccination or protein-replacement therapies.
Contributions addressing fundamental or clinical researches dealing with nucleic acids-based strategies from oligonucleotides to mRNAs and DNAs, as well as key issues on viral or non-viral delivery vectors will be welcomed.
Cell therapy-based strategies also offer a great potential for the treatment of human diseases. As an example, pluripotent stem cells can be generated from somatic cells with the capacity to proliferate indefinitely and to be reprogrammed for differentiation at will. Challenges dealing with tumorigenicity and immunogenicity have had to be considered, which has delayed the onset of clinical trials until recently.
Contributions dealing with various aspects and applications of cell-based strategies will be welcomed, from animal models to human diseases clinical studies.
Dr. Bernard Lebleu
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