Gene editing as a promising approach for respiratory diseases

Bai, Yichun; Liu, Yang; Su, Zhenlei; Ma, Yana; Ren, Chonghua; Zhao, Runzhen; Ji, Hong‐Long

doi:10.1136/jmedgenet-2017-104960

Cited by 8 publications

(6 citation statements)

References 93 publications

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“…Two methods commonly used to suppress the expression of a specific protein are genome editing [mainly CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR‐associated 9) technology] and RNAi. CRISPR/Cas9 has been developed in the last few years and has been successfully used for different research purposes in different systems (Bai et al , ; Bier et al , ; Kell et al , ). CRISPR/Cas9 has a number of advantages, including the generation of complete knockouts without necessarily transgenic sequences present in the final mutant.…”

Section: Resultsmentioning

confidence: 99%

DCL‐suppressed Nicotiana benthamiana plants: valuable tools in research and biotechnology

Κατσαρού

Mitta

Bardani

et al. 2018

Molecular Plant Pathology

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Summary RNA silencing is a universal mechanism involved in development, epigenetic modifications and responses to biotic and abiotic stresses. The major components of this mechanism are Dicer‐like (DCL), Argonaute (AGO) and RNA‐dependent RNA polymerase (RDR) proteins. Understanding the role of each component is of great scientific and agronomic importance. Plants, including Nicotiana benthamiana , an important plant model, usually possess four DCL proteins, each of which has a specific role, namely being responsible for the production of an exclusive small RNA population. Here, we used RNA interference (RNAi) technology to target DCL proteins and produced single and combinatorial mutants for DCL. We analysed the phenotype for each DCL knockdown plant, together with the small RNA profile, by next‐generation sequencing (NGS). We also investigated transgene expression, as well as viral infections, and were able to show that DCL suppression results in distinct developmental defects, changes in small RNA populations, increases in transgene expression and, finally, higher susceptibility in certain RNA viruses. Therefore, these plants are excellent tools for the following: (i) to study the role of DCL enzymes; (ii) to overexpress proteins of interest; and (iii) to understand the complex relationship between the plant silencing mechanism and biotic or abiotic stresses.

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Section: Resultsmentioning

confidence: 99%

DCL‐suppressed Nicotiana benthamiana plants: valuable tools in research and biotechnology

Κατσαρού

Mitta

Bardani

et al. 2018

Molecular Plant Pathology

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“…Respiratory diseases seriously affect the physical and mental well-being of patients with symptoms of sneezing, cough, and difficulty in breathing, which are leading causes of mortality and morbidity [ 76 ]. The natural botanical materials from Fritillaria have significant pharmacological effects on the respiratory system, including the alleviation of cough, phlegm, asthma, COPD, and acute lung injury (ALI) (Fig.…”

Section: Pharmacological Activitiesmentioning

confidence: 99%

Natural drug sources for respiratory diseases from Fritillaria: chemical and biological analyses

et al. 2021

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Fritillaria naturally grows in the temperate region of Northern Hemisphere and mainly distributes in Central Asia, Mediterranean region, and North America. The dried bulbs from a dozen species of this genus have been usually used as herbal medicine, named Beimu in China. Beimu had rich sources of phytochemicals and have extensively applied to respiratory diseases including coronavirus disease (COVID-19). Fritillaria species have alkaloids that act as the main active components that contribute multiple biological activities, including anti-tussive, expectorant, and anti-asthmatic effects, especially against certain respiratory diseases. Other compounds (terpenoids, steroidal saponins, and phenylpropanoids) have also been identified in species of Fritillaria. In this review, readers will discover a brief summary of traditional uses and a comprehensive description of the chemical profiles, biological properties, and analytical techniques used for quality control. In general, the detailed summary reveals 293 specialized metabolites that have been isolated and analyzed in Fritillaria species. This review may provide a scientific basis for the chemical ecology and metabolomics in which compound identification of certain species remains a limiting step.

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“…CRISPR‐mediated gene correction has several advantages over conventional gene therapy for the treatment of CF and other congenital diseases. Genetic modifications induced by CRISPR are maintained permanently in edited cells, and corrected genes remain under the control of their endogenous promoter . Unlike cDNA gene therapy, the specific method of CRISPR gene editing must be adapted according to the nature of the mutation being corrected.…”

Section: Crispr/cas Gene Therapy For Respiratory Diseasesmentioning

confidence: 99%

“…Congenital lung diseases including surfactant protein deficiency, cystic fibrosis (CF), alpha‐1 antitrypsin (AAT) deficiency and other rare monogenic diseases occur with less frequency, but also have significant impact on patient health and lifespan . Causes of this diverse set of diseases include environmental factors such as smoking, air pollution and infection, as well as somatic or inherited genetic mutations and ageing . As more is known about the genetic factors that contribute to respiratory disease, there is a need for novel tools that can manipulate the sequence and expression of endogenous genes.…”

Section: Introductionmentioning

confidence: 99%

Applications of CRISPR systems in respiratory health: Entering a new ‘red pen’ era in genome editing

Moses

Kaur

2019

Respirology

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Respiratory diseases, such as influenza infection, acute tracheal bronchitis, pneumonia, tuberculosis, chronic obstructive pulmonary disease, asthma, lung cancer and nasopharyngeal carcinoma, continue to significantly impact human health. Diseases of the lung and respiratory tract are influenced by environmental conditions and socio-economic factors; however, many of these serious respiratory disorders are also rooted in genetic or epigenetic causes. Clustered regularly interspaced palindromic repeats (CRISPR) and CRISPRassociated (Cas) proteins, isolated from the immune system of prokaryotes, provide a tool to manipulate gene sequences and gene expression with significant implications for respiratory research. CRISPR/Cas systems allow preclinical modelling of causal factors involved in many respiratory diseases, providing new insights into their underlying mechanisms. CRISPR can also be used to screen for genes involved in respiratory processes, development and pathology, identifying novel disease drivers or drug targets. Finally, CRISPR/-Cas systems can potentially correct genetic mutations and edit epigenetic marks that contribute to respiratory disorders, providing a form of personalized medicine that could be used in conjunction with other technologies such as stem cell reprogramming and transplantation. CRISPR gene editing is a young field of research, and concerns regarding its specificity, as well as the need for efficient and safe delivery methods, need to be addressed further. However, CRISPR/Cas systems represent a significant step forward for research and therapy in respiratory health, and it is likely we will see the breakthroughs generated from this technology continue.

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Gene editing as a promising approach for respiratory diseases

Cited by 8 publications

References 93 publications

DCL‐suppressed Nicotiana benthamiana plants: valuable tools in research and biotechnology

DCL‐suppressed Nicotiana benthamiana plants: valuable tools in research and biotechnology

Natural drug sources for respiratory diseases from Fritillaria: chemical and biological analyses

Applications of CRISPR systems in respiratory health: Entering a new ‘red pen’ era in genome editing

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