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Cells
Special Issues
Advances and Breakthroughs in Stem Cell Research
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Advances and Breakthroughs in Stem Cell Research

A special issue of Cells (ISSN 2073-4409). This special issue belongs to the section "Stem Cells".

Deadline for manuscript submissions: 31 August 2024 | Viewed by 269

Special Issue Editors

Prof. Dr. Alexander E. Kalyuzhny

E-Mail Website
Guest Editor
Neuroscience, UMN Twin Cities, 6-145 Jackson Hall, 321 Church St SE, Minneapolis, MN 55455, USA
Interests: investigating the mechanisms underlying the constitutive induced heteromerization of opioid receptors
Special Issues, Collections and Topics in MDPI journals
Dr. Hemant Kumar Mishra

E-Mail
Guest Editor
Cellinfinitybio, San Francisco, CA, USA
Interests: induced pluripotent stem cell-derived NK and T cells; hematopoietic stem cells; cancer immunotherapy; therapeutic antibody; host-pathogen interaction; immune dysfunction; off the shelf therapy; checkpoint inhibitors; metalloproteases; small molecule inhibitors; cardiovascular disorders; tuberculosis; sarcoidosis; allergy and asthma

Special Issue Information

Dear Colleagues,

This Special Issue will center around the latest cutting-edge breakthroughs in stem cell research within immunotherapy and regenerative medicine. In recent years, advancements in stem cell research have opened promising avenues for treating and potentially curing diverse conditions such as cancers, metabolic disorders, and neurodegenerative diseases. The immense potential of specific human iPSC-derived cell lineages, including CAR T cells, CAR NK cells, neurons, cardiomyocytes, and pancreatic islets, holds transformative possibilities in revolutionizing medical practices through the development of innovative therapies. This Special Issue will explore a range of topics related to stem cell therapy, encompassing iPSC (induced pluripotent stem cell)- or hematopoietic-stem-cell-derived, therapeutically relevant lineages. We welcome original manuscripts and reviews that delve into these subjects, contributing to a more profound comprehension of recent advancements and challenges in these domains.

Prof. Dr. Alexander E. Kalyuzhny
Dr. Hemant Kumar Mishra
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. Cells is an international peer-reviewed open access semimonthly 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 2700 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

  • stem cell
  • induced pluripotent stem cell
  • iPSC
  • hematopoietic stem cell

Published Papers (1 paper)

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Research

17 pages, 26759 KiB  
Article
A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs
by Neha R. Dhoke, Hyunkee Kim, Karim Azzag, Sarah B. Crist, James Kiley and Rita C. R. Perlingeiro
Cells 2024, 13(11), 972; https://doi.org/10.3390/cells13110972 - 4 Jun 2024
Abstract
Mutations in the DMD gene cause fatal Duchenne Muscular Dystrophy (DMD). An attractive therapeutic approach is autologous cell transplantation utilizing myogenic progenitors derived from induced pluripotent stem cells (iPSCs). Given that a significant number of DMD mutations occur between exons 45 and 55, [...] Read more.
Mutations in the DMD gene cause fatal Duchenne Muscular Dystrophy (DMD). An attractive therapeutic approach is autologous cell transplantation utilizing myogenic progenitors derived from induced pluripotent stem cells (iPSCs). Given that a significant number of DMD mutations occur between exons 45 and 55, we developed a gene knock-in approach to correct any mutations downstream of exon 44. We applied this approach to two DMD patient-specific iPSC lines carrying mutations in exons 45 and 51 and confirmed mini-DYSTROPHIN (mini-DYS) protein expression in corrected myotubes by western blot and immunofluorescence staining. Transplantation of gene-edited DMD iPSC-derived myogenic progenitors into NSG/mdx4Cv mice produced donor-derived myofibers, as shown by the dual expression of human DYSTROPHIN and LAMIN A/C. These findings further provide proof-of-concept for the use of programmable nucleases for the development of autologous iPSC-based therapy for muscular dystrophies. Full article
(This article belongs to the Special Issue Advances and Breakthroughs in Stem Cell Research)
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