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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
Current status and future directions of gene and cell therapy for cystic fibrosis
Uta Griesenbach1, Eric W F W Alton
1Department of Gene Therapy, Imperial College London, London, UK. u.griesenbach@imperial.ac.uk
Abstract:
Although the development of gene therapy for cystic fibrosis (CF) was high priority for many groups in academia and industry in the first 10-15 years after cloning the cystic fibrosis transmembrane conductance regulator (CFTR) gene, more recently active research into CF gene therapy is only being performed by a small number of committed groups. However, despite the waning enthusiasm, which is largely due to the realization that gene transfer into lungs is more difficult than originally thought, and the fact that meaningful clinical trials are expensive and difficult to perform, gene therapy continues to hold promise for the treatment of CF lung disease. Problems related to repeat administration of adenovirus and adeno-associated virus-based vectors led to a focus on non-viral vectors in clinical trials. However, the recent evidence that lentiviral vectors may be able to evade the immune system and, thereby, allow for repeat administration and long-lasting expression opens new doors for the use of viral vectors in the context of CF gene therapy. In addition, early pre-clinical studies have recently been initiated to address cell therapy-based approaches for CF. In this review, we discuss recent developments with viral and non-viral vectors and cell therapy, and provide an update on clinical gene therapy studies.
Insights
Gene therapy for cystic fibrosis (CF) faces challenges but shows promise. Recent advances in viral vectors, non-viral vectors, and cell therapy offer new hope for treating CF lung disease.
Area of Science:
- Pulmonary Medicine
- Genetics
- Biotechnology
Background:
- Gene therapy for cystic fibrosis (CF) was a high priority after cloning the CFTR gene.
- Research has slowed due to difficulties in lung gene transfer and high costs of clinical trials.
- Despite challenges, gene therapy remains a promising treatment for CF lung disease.
Purpose of the Study:
- To review recent developments in gene therapy for cystic fibrosis.
- To update on clinical gene therapy studies, viral and non-viral vectors, and cell therapy approaches.
- To highlight new possibilities for CF treatment.
Main Methods:
- Review of recent advancements in viral vectors (adenovirus, AAV, lentiviral) for gene transfer.
- Discussion of non-viral vectors and their use in clinical trials.
- Exploration of emerging cell therapy-based approaches for CF.
Main Results:
- Lentiviral vectors show potential to evade immune responses, enabling repeat administration and long-lasting expression.
- Non-viral vectors are being explored in clinical trials due to issues with repeat administration of viral vectors.
- Early pre-clinical studies for cell therapy-based approaches in CF have been initiated.
Conclusions:
- Gene therapy for CF continues to evolve, with lentiviral vectors offering new avenues for viral vector-based treatments.
- Non-viral vectors and cell therapies represent important alternative and complementary strategies.
- Ongoing research and clinical trials are crucial for realizing the full potential of gene therapy in treating cystic fibrosis.
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