The Evolving Landscape of Cell and Gene Therapy—Opportunities, Challenges and the Road Ahead

A special issue of Cells (ISSN 2073-4409).

Deadline for manuscript submissions: 15 July 2026 | Viewed by 2468

Editor


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Guest Editor
1. MirnaCures Therapeutics, Boston, MA, USA
2. College of Professional Studies, Northeastern University, Boston, MA 02101-02117, USA
Interests: cell and gene therapy; rare diseases; cardiovascular; metabolic disease; atherosclerosis; dyslipidemia; animal models; drug discovery and development
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Special Issue Information

Dear Colleagues,

Cell and gene therapies (CGT) have emerged as powerful modalities for treating conditions once considered intractable. With over 22 approved therapies to date and hundreds more in development, the field is undergoing rapid and transformative growth. Significant strides have been made in improving targeted delivery and reducing off-target effects, allowing for greater precision and safety in clinical applications.

Cell therapies—such as CAR-T cells—restore or enhance cellular functions by introducing viable, functional cells into patients. Gene therapies, in contrast, aim to correct or silence defective genes through direct modification of genetic material. Together, these approaches have expanded therapeutic possibilities for rare genetic disorders, cancers, and neurodegenerative diseases—particularly in patients who fail to respond to standard treatments or do not reach therapeutic targets.

This Special Issue focuses on the recent advances in CGT, with particular attention to addressing unmet medical needs over the past decade. It also highlights the critical role of in vitro and in vivo models in optimizing therapeutic strategies and the emerging potential to target previously inaccessible tissues and molecular pathways.

As we enter a new era of personalized and precision medicine, it is imperative to critically evaluate both the promise and limitations of CGT. This issue aims to foster dialogue and innovation toward more effective, scalable, and equitable therapeutic solutions.

Dr. Rai Ajit K. Srivastava
Guest Editor

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Keywords

  • cell therapy
  • gene therapy/gene editing
  • micro RNAs
  • rare diseases
  • metabolic disease
  • cancer
  • diabetes
  • proatherogenic lipoproteins
  • drug discovery
  • ASO

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Published Papers (1 paper)

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Review

42 pages, 993 KB  
Review
CRISPR–Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics
by Adrianna Rutkowska, Tadeusz Strózik, Tomasz Wasiak, Damian Ciunowicz, Natalia Kapelan, Natalia Szczepaniak, Juliusz Sosnowski, Weronika Goślińska, Jakub Bartkowiak, Agata Budny-Lewandowska, Patrycja Antończyk, Maria Markiewicz, Piotr Gustaw, Kamil Filiks, Maria Jaskólska and Ewelina Stoczyńska-Fidelus
Cells 2026, 15(7), 644; https://doi.org/10.3390/cells15070644 - 2 Apr 2026
Viewed by 1982
Abstract
CRISPR–Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used [...] Read more.
CRISPR–Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used across early CRISPR clinical translation. In parallel, early clinical experience has begun to demonstrate the feasibility of in vivo editing, including subretinal delivery for CEP290-associated inherited retinal degeneration (EDIT-101 programme) and hepatocyte-targeted lipid nanoparticles (LNPs) for liver-derived targets such as transthyretin and plasma prekallikrein (KLKB1). As translation expands across hematologic, metabolic, ocular and oncology indications, development is increasingly constrained by the predictability and safety of editing outcomes, delivery-determined biodistribution and exposure time, and immune recognition of bacterial Cas9 orthologs and delivery components. We summarize diagnostic readouts for confirming patient genotype, quantifying on-target editing and expression changes, assessing off-target and structural outcomes using orthogonal assays, and monitoring clonal dynamics and immune responses during long-term follow-up. We also discuss how these readouts interface with CMC controls and regulatory expectations for advanced therapy medicinal products (ATMPs), highlighting the need for fit-for-purpose, standardized testing frameworks in early trials. Full article
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