ijms-logo

Journal Browser

Journal Browser

Genome Editing in Autologous Stem Cells: From Bench to Bedside

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Genetics and Genomics".

Deadline for manuscript submissions: 20 April 2026 | Viewed by 525

Special Issue Editor


E-Mail Website
Guest Editor
School of Medicine, Stanford University, Stanford, CA 94305, USA
Interests: genome editing; gene therapy; lysosomal diseases; rare diseases; hematopoietic stem cells; stem cells; mucopolysaccharidosis

Special Issue Information

Dear Colleagues,

Precise genome editing technologies have revolutionized the field of regenerative medicine, enabling autologous stem cell transplantation as a potential therapeutic option for multiple diseases. These emerging therapies offer a promising alternative to allogeneic transplantation, eliminating the need for donors and minimizing immune complications.

We are pleased to invite you to contribute to our Special Issue, “Genome Editing in Autologous Stem Cells: From Bench to Bedside”. This Special Issue aims to highlight recent advances and ongoing challenges in translating the genome editing of autologous stem cells from the research lab to clinical application.

We welcome original research articles, reviews, commentaries, and short communications. Relevant research areas include (but are not limited to) the following:

  • Preclinical studies: genome editing tools, delivery systems, and new strategies to improve editing efficiency in stem cells; preclinical models and proof-of-concept studies for disease correction;
  • Clinical product development: process development, scalability, quality control, chemistry, manufacturing, and controls (CMC), and regulatory pathways;
  • Safety: safety assessments, off-target analyses, and regulatory requirements;
  • Ethics: ethical, economic, and accessibility issues with autologous genome-edited products.

We look forward to receiving your contributions.

Dr. Edina Poletto
Guest Editor

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 250 words) can be sent to the Editorial Office for assessment.

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. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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
  • gene therapy
  • autologous transplant
  • preclinical studies
  • process development
  • scalability
  • quality control
  • manufacturing
  • regulatory compliance
  • IND-enabling studies

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.
  • Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.

Further information on MDPI's Special Issue policies can be found here.

Published Papers (1 paper)

Order results
Result details
Select all
Export citation of selected articles as:

Review

20 pages, 1067 KB  
Review
Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies
by Karen O’Hanlon Cohrt and Shirley O’Dea
Int. J. Mol. Sci. 2026, 27(8), 3384; https://doi.org/10.3390/ijms27083384 - 9 Apr 2026
Viewed by 251
Abstract
Allogeneic hematopoietic cell transplantation (HCT) has been used for decades to treat certain malignant and non-malignant hematological conditions, but challenges remain. Increased understanding of disease mechanisms and recent developments in genome editing have enabled alternative strategies utilizing gene-edited autologous HCT and many of [...] Read more.
Allogeneic hematopoietic cell transplantation (HCT) has been used for decades to treat certain malignant and non-malignant hematological conditions, but challenges remain. Increased understanding of disease mechanisms and recent developments in genome editing have enabled alternative strategies utilizing gene-edited autologous HCT and many of these have progressed to the clinic. We present here a comprehensive review of clinical trials of gene-edited autologous hematopoietic stem cells for the treatment of hemoglobinopathies and immunodeficiencies. Searches of major international clinical trial registries were carried out using specific key words. In total, 44 interventional clinical trials investigating gene-edited autologous stem cell therapies were identified, with CASGEVY (exagamglogene autotemcel) being the only product approved to date. Hemoglobinopathies were the most common indication (n = 37) followed by immunodeficiencies (n = 4), with single trials in HIV-1 infection, pyruvate kinase deficiency and limb–girdle muscular dystrophy. Gene-editing strategies fall into three categories: disruption of the BCL11A erythroid enhancer, editing of the γ-globin promoter and direct correction or disruption of disease-relevant genes. CD34+ hematopoietic stem and progenitor cells are the most common cell types edited, and CRISPR-Cas9 is the most widely used gene-editing modality. While results are encouraging, efficient intracellular delivery of gene-editing tools, editing efficiencies and off-target editing remain challenges for the field. Full article
(This article belongs to the Special Issue Genome Editing in Autologous Stem Cells: From Bench to Bedside)
Show Figures

Figure 1

Back to TopTop