Research Topic Highlights
The article collection explores advances in gene editing and stem cell therapies targeting respiratory diseases caused by genetic mutations and chemical injuries. It highlights critical progress made in understanding and treating genetic pulmonary conditions such as severe respiratory distress syndrome (RDS), childhood interstitial lung disease (chILD), and cystic fibrosis (CF). The reviewed research emphasizes innovative techniques employing gene editing tools, including CRISPR/Cas9, homology-directed repair (HDR), and intrachromosomal homologous recombination (IHR), alongside the use of stem cell interventions aimed at airway epithelium regeneration. Specific pathogenic variants associated with surfactant dysfunction (SFTPB, SFTPC, ABCA3) and mutations in the CFTR gene are discussed in relation to their clinical manifestations and potential interventions. Furthermore, approaches to create universal donor stem cells (UDSC) to treat inhalation injuries from vesicants such as sulfur mustard and efforts in achieving seamless and mutation-specific genetic corrections in induced pluripotent stem cells (iPSC) provide comprehensive insight into personalized and universal therapeutic strategies. This collection illustrates both the promising outcomes and significant challenges in developing safe, efficient, and precise gene and cell-based therapies aimed at restoring respiratory function and improving patient outcomes in severe pulmonary disorders.
Context and Scope
Recent advances in CRISPR/Cas9 and other genome modification systems resulted in the development of therapeutics for many monogenic diseases – especially those affecting the hematopoietic system. Cystic fibrosis (CF) is one of the most common genetic diseases that affects many organ systems but causes significant damage to the lungs. Progress in gene therapy and genome editing approaches to treat CF has been particularly challenging due to difficulties in targeting and editing airway stem cells, delivering genome editing systems into the airways and transplanting airway stem cells. Recent studies have reported the correction of CF causing mutations in airway stem cells, embryonic stem cells, induced pluripotent stem cells and intestinal stem cells using CRISPR/Cas9 and other related tools such as base editing and prime editing. In addition to the correction of CF causing mutations ex vivo, attempts have also been made to deliver genome editing reagents and correct mutations causing CF and other airway diseases both in vivo and in utero.
Multiple questions remain to be answered in our efforts to develop an effective gene correction strategy to treat CF and other genetic diseases affecting the airways. In this Research Topic, we will present studies that advance the use of genome editing to treat CF and other diseases affecting the airways. These articles will focus on the genome modification of airway stem cells, delivery of genome editing reagents into the airways and approaches to correct CF causing mutations. We will also present reviews/perspectives on the development of gene therapy and genome editing for CF and other airway diseases.
We encourage submissions in the form of Original Research, Methods, Reviews, Mini Review, Opinion, and Hypothesis and Theory. Suitable topics include, but are not limited to:
· Genome editing (Homologous recombination (HR) or non-homologous end-joining (NHEJ) based) to correct or model CF causing mutations in any cell type
· Genome editing (HR or NHEJ based) based strategy to treat any airway disease
· Base and Prime Editing tools targeting CF in any organ or other airway diseases
· Enhancement/optimization of current approaches to precise gene editing in airway cells
· Understanding of homology directed repair (HDR) mechanisms in the context of gene correction and gene addition in airway cells
· In vivo delivery of genome editing reagents (e.g. Cas9 RNP, mRNA, other nucleases) to the airways
Research Topic Highlights
The article collection explores advances in gene editing and stem cell therapies targeting respiratory diseases caused by genetic mutations and chemical injuries. It highlights critical progress made in understanding and treating genetic pulmonary conditions such as severe respiratory distress syndrome (RDS), childhood interstitial lung disease (chILD), and cystic fibrosis (CF). The reviewed research emphasizes innovative techniques employing gene editing tools, including CRISPR/Cas9, homology-directed repair (HDR), and intrachromosomal homologous recombination (IHR), alongside the use of stem cell interventions aimed at airway epithelium regeneration. Specific pathogenic variants associated with surfactant dysfunction (SFTPB, SFTPC, ABCA3) and mutations in the CFTR gene are discussed in relation to their clinical manifestations and potential interventions. Furthermore, approaches to create universal donor stem cells (UDSC) to treat inhalation injuries from vesicants such as sulfur mustard and efforts in achieving seamless and mutation-specific genetic corrections in induced pluripotent stem cells (iPSC) provide comprehensive insight into personalized and universal therapeutic strategies. This collection illustrates both the promising outcomes and significant challenges in developing safe, efficient, and precise gene and cell-based therapies aimed at restoring respiratory function and improving patient outcomes in severe pulmonary disorders.
Context and Scope
Recent advances in CRISPR/Cas9 and other genome modification systems resulted in the development of therapeutics for many monogenic diseases – especially those affecting the hematopoietic system. Cystic fibrosis (CF) is one of the most common genetic diseases that affects many organ systems but causes significant damage to the lungs. Progress in gene therapy and genome editing approaches to treat CF has been particularly challenging due to difficulties in targeting and editing airway stem cells, delivering genome editing systems into the airways and transplanting airway stem cells. Recent studies have reported the correction of CF causing mutations in airway stem cells, embryonic stem cells, induced pluripotent stem cells and intestinal stem cells using CRISPR/Cas9 and other related tools such as base editing and prime editing. In addition to the correction of CF causing mutations ex vivo, attempts have also been made to deliver genome editing reagents and correct mutations causing CF and other airway diseases both in vivo and in utero.
Multiple questions remain to be answered in our efforts to develop an effective gene correction strategy to treat CF and other genetic diseases affecting the airways. In this Research Topic, we will present studies that advance the use of genome editing to treat CF and other diseases affecting the airways. These articles will focus on the genome modification of airway stem cells, delivery of genome editing reagents into the airways and approaches to correct CF causing mutations. We will also present reviews/perspectives on the development of gene therapy and genome editing for CF and other airway diseases.
We encourage submissions in the form of Original Research, Methods, Reviews, Mini Review, Opinion, and Hypothesis and Theory. Suitable topics include, but are not limited to:
· Genome editing (Homologous recombination (HR) or non-homologous end-joining (NHEJ) based) to correct or model CF causing mutations in any cell type
· Genome editing (HR or NHEJ based) based strategy to treat any airway disease
· Base and Prime Editing tools targeting CF in any organ or other airway diseases
· Enhancement/optimization of current approaches to precise gene editing in airway cells
· Understanding of homology directed repair (HDR) mechanisms in the context of gene correction and gene addition in airway cells
· In vivo delivery of genome editing reagents (e.g. Cas9 RNP, mRNA, other nucleases) to the airways
