Genome-Edited Adult Stem Cells: Next-Generation Advanced Therapy Medicinal Products
A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".
Deadline for manuscript submissions: closed (20 February 2023) | Viewed by 4809
Special Issue Editors
Interests: CAR-T cells; gene therapy; cancer immunotherapy; T cells; hematopoietic stem cells; gene editing
Interests: gene therapy; genome editing; cancer immunotherapy; T cells; hematopoietic stem cells; rare diseases
Special Issue Information
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
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.
Keywords
- 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
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.
Further information on MDPI's Special Issue policies can be found here.
