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Pharmaceutics
Special Issues
New Strategies in Gene and Cell Therapy for Neurological Disorders
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New Strategies in Gene and Cell Therapy for Neurological Disorders

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: 30 November 2026 | Viewed by 4097

Special Issue Editor

Dr. Jubayer Hossain

E-Mail Website
Guest Editor
Faculty of Nursing and Health Sciences, Nord University, Bodø, Norway
Interests: cancer; glioblastoma; gene therapy; cell therapy; immunotherapy; lentiviral vectors

Special Issue Information

Dear Colleagues,

Neurological disorders encompass a diverse spectrum of conditions affecting the central and peripheral nervous systems, including neurodegenerative diseases, neurodevelopmental disorders, neuropsychiatric conditions, genetic and metabolic disorders, CNS tumors, and trauma-induced injuries. Gene and cell therapy holds immense promise for treating these disorders, offering potential solutions for previously intractable conditions. Recent advancements in gene editing technologies, vector systems, cell-based regenerative medicine, immunomodulatory therapies, and delivery strategies have opened up new avenues for therapeutic interventions.

This Special Issue aims to feature cutting-edge gene and cell therapy research across the broad landscape of neurological disorders. Special attention will be paid to emerging technologies, including, but not limited to, nanotechnology, novel vectors, innovative delivery strategies to overcome the blood–brain barrier, and immunomodulatory interventions. Furthermore, reports on clinical trials or regulatory aspects will also be paid similar attention. This Special Issue welcomes the submission of original research articles demonstrating novel approaches, comprehensive reviews synthesizing current knowledge, and perspective pieces discussing future directions.

By bringing together diverse expertise in gene and cell therapy, neuroscience, and clinical translation, this Special Issue aims to accelerate the development of more effective treatments for neurological disorders.

We are pleased to invite you to submit original research articles, reviews, and case studies that address the aforementioned topics. Submissions should provide new insights, present innovative methodologies, or offer comprehensive reviews of current knowledge.

This Special Issue aims to place a spotlight on both breakthrough advances and critical challenges in gene and cell therapy for neurological disorders, encompassing neurodevelopmental, neurodegenerative, and neuropsychiatric disorders, CNS tumors, CNS injury, and rare genetic neurological diseases. By bringing together cutting-edge research, we seek to provide a comprehensive overview of current trends, technological limitations, safety considerations, and promising solutions in therapeutic interventions. Special emphasis will be placed on identifying key bottlenecks and innovative approaches that could transform our understanding and implementation of these therapeutic strategies.

We look forward to receiving your contributions.

Dr. Jubayer Hossain
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. 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

  • gene and cell therapy
  • neurodegenerative diseases
  • neurodevelopmental disorders
  • neuropsychiatric conditions
  • CNS tumors
  • trauma and injury in the CNS
  • blood–brain barrier
  • neuroimmunology and immunomodulation
  • peripheral nervous system disorders
  • regenerative medicine

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Published Papers (2 papers)

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11 pages, 5225 KB  
Article
Tropism Profiling of Lentiviral Vector Pseudotypes in Diverse Brain Tumor Models
by Johannes K. Andersen, Lars A. R. Ystaas, Rolf Bjerkvig, Hrvoje Miletic and Jubayer A. Hossain
Pharmaceutics 2026, 18(1), 137; https://doi.org/10.3390/pharmaceutics18010137 - 22 Jan 2026
Viewed by 600
Abstract
Background: Lentiviral vectors (LVs) show promise as gene therapy tools for brain tumors, but optimal envelope protein choices for different tumor types have not been determined. Methodology: This study evaluated three pseudotyped LV variants—VSV-GP, FuG-B2, and LCMV-GP—across diverse brain tumor cell [...] Read more.
Background: Lentiviral vectors (LVs) show promise as gene therapy tools for brain tumors, but optimal envelope protein choices for different tumor types have not been determined. Methodology: This study evaluated three pseudotyped LV variants—VSV-GP, FuG-B2, and LCMV-GP—across diverse brain tumor cell lines including glioblastoma (GBM), diffuse intrinsic pontine glioma (DIPG), medulloblastoma, and metastatic brain cancers. Results: VSV-GP and FuG-B2 pseudotypes significantly outperformed LCMV-GP across most tumor types. Both VSV-GP and FuG-B2 demonstrated high transduction efficiency in GBM and DIPG cells, though some cell lines displayed selective preferences for one pseudotype over the other. Medulloblastoma cells were challenging to transduce, with only VSV-GP achieving substantial efficacy. Metastatic brain cancers showed distinct tropism patterns: melanoma metastases were preferentially transduced by the FuG-B2 pseudotype, while lung metastases showed preference for the VSV-GP pseudotype. Conclusions: These findings suggest envelope protein selection should be tailored to specific brain tumor types. VSV-GP appears most suitable for medulloblastoma and lung metastases, FuG-B2 for melanoma metastases, and both for GBM and DIPG gene therapy applications. The study provides crucial guidance for translating lentiviral gene therapy to clinical applications, supporting personalized treatment strategies based on tumor-specific vector tropism profiles. Full article
(This article belongs to the Special Issue New Strategies in Gene and Cell Therapy for Neurological Disorders)
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25 pages, 13483 KB  
Article
Targeting TDP-43 Proteinopathy in hiPSC-Derived Mutated hNPCs with Mitoxantrone Drugs and miRNAs
by Uzair A. Ansari, Ankita Srivastava, Ankur K. Srivastava, Abhishek Pandeya, Pankhi Vatsa, Renu Negi, Akash Singh and Aditya B. Pant
Pharmaceutics 2025, 17(4), 410; https://doi.org/10.3390/pharmaceutics17040410 - 25 Mar 2025
Viewed by 3005
Abstract
Background/Objectives: TDP-43 mutation-driven Amyotrophic Lateral Sclerosis (ALS) motor neuron disease is one of the most prominent forms (approximately 97%) in cases of sporadic ALS. Dysfunctional autophagy and lysosomal function are the prime mechanisms behind ALS. Mitoxantrone (Mito), a synthetic doxorubicin analog, is an [...] Read more.
Background/Objectives: TDP-43 mutation-driven Amyotrophic Lateral Sclerosis (ALS) motor neuron disease is one of the most prominent forms (approximately 97%) in cases of sporadic ALS. Dysfunctional autophagy and lysosomal function are the prime mechanisms behind ALS. Mitoxantrone (Mito), a synthetic doxorubicin analog, is an inhibitor of DNA and RNA synthesis/repair via intercalating with nitrogenous bases and inhibiting topoisomerase II. The therapeutic potential of miRNAs associated with disease conditions has also been reported. This study explores the therapeutic potential of Mito along with miRNAs against mutated TDP-43 protein-induced proteinopathy in human-induced pluripotent stem cell (hiPSC)-derived human neural progenitor cells (hNPCs). Methods: HiPSCs mutated for TDP-43 were differentiated into hNPCs and used to explore the therapeutic potential of Mito at a concentration of 1 μM for 24 h (the identified non-cytotoxic dose). The therapeutic effects of Mito on miRNA expression and various cellular parameters such as mitochondrial dynamics, autophagy, and stress granules were assessed using the high-throughput Open Array technique, immunocytochemistry, flow cytometry, immunoblotting, and mitochondrial bioenergetic assay. Results: Mutated TDP-43 protein accumulation causes stress granule formation (G3BP1), mitochondrial bioenergetic dysfunction, SOD1 accumulation, hyperactivated autophagy, and ER stress in hNPCs. The mutated hNPCs also show dysregulation in six miRNAs (miR-543, miR-34a, miR-200c, miR-22, miR-29b, and miR-29c) in mutated hNPCs. A significant restoration of TDP-43 mutation-induced alterations could be witnessed upon the exposure of mutated hNPCs to Mito. Conclusions: Our study indicates that miR-543, miR-29b, miR-22, miR-200c, and miR-34a have antisense therapeutic potential alone and in combination with Mitoxantrone. Full article
(This article belongs to the Special Issue New Strategies in Gene and Cell Therapy for Neurological Disorders)
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