Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Lee, Bumwhee; Lee, Kunwoo; Panda, Shree; Gonzales‐Rojas, Rodrigo; Chong, Anthony T; Bugay, Vladislav; Park, Hyo Min; Brenner, Robert; Murthy, Niren; Lee, Hye Young

doi:10.1038/s41551-018-0252-8

Cited by 287 publications

(186 citation statements)

References 54 publications

(53 reference statements)

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“…Furthermore, the marble‐burying assay and the empty cage observation test were implemented in both wild‐type mice and Fmr1 knockout mice to evaluate the repetitive behaviors. It was found that the symptoms of exaggerated repetitive behaviors were alleviated after the administration of CRISPR‐Gold nanoparticles (Figure C, D) …”

Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 97%

“…(D) mGluR5‐CRISPR successfully promotes mGluR5 gene editing in the striatum of wild‐type and Fmr1 knockout mice. Reproduced with permission…”

Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 99%

“…The mGluR5 gene served as a target for CRISPR editing with respect to treatment of FXS, because of the exaggerated mGluR5 signaling is closely related to the FXS pathophysiology and other ASDs. To treat FXS, Lee et al . used CRISPR‐Gold nanocomplexes to deliver Cas9 into the striatum of adult mice brain via local injection.…”

Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 99%

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Delivery of CRISPR/Cas9 for therapeutic genome editing

Wan

Xin

et al. 2019

The Journal of Gene Medicine

101

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The clustered, regularly‐interspaced, short palindromic repeat (CRISPR)‐associated nuclease 9 (CRISPR/Cas9) is emerging as a promising genome‐editing tool for treating diseases in a precise way, and has been applied to a wide range of research in the areas of biology, genetics, and medicine. Delivery of therapeutic genome‐editing agents provides a promising platform for the treatment of genetic disorders. Although viral vectors are widely used to deliver CRISPR/Cas9 elements with high efficiency, they suffer from several drawbacks, such as mutagenesis, immunogenicity, and off‐target effects. Recently, non‐viral vectors have emerged as another class of delivery carriers in terms of their safety, simplicity, and flexibility. In this review, we discuss the modes of CRISPR/Cas9 delivery, the barriers to the delivery process and the application of CRISPR/Cas9 system for the treatment of genetic disorders. We also highlight several representative types of non‐viral vectors, including polymers, liposomes, cell‐penetrating peptides, and other synthetic vectors, for the therapeutic delivery of CRISPR/Cas9 system. The applications of CRISPR/Cas9 in treating genetic disorders mediated by the non‐viral vectors are also discussed.

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Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 97%

“…(D) mGluR5‐CRISPR successfully promotes mGluR5 gene editing in the striatum of wild‐type and Fmr1 knockout mice. Reproduced with permission…”

Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 99%

Section: Non‐viral Vectors Of Crispr‐cas9mentioning

confidence: 99%

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Delivery of CRISPR/Cas9 for therapeutic genome editing

Wan

Xin

et al. 2019

The Journal of Gene Medicine

101

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“…Researchers have used CRISPR‐based editing to correct the genetic basis of many diseases in isolated cells or animal models . The first wave of clinical trials using CRISPR enzymes to treat inherited disorders in humans involve removing a patient's cells, editing ex vivo, and reinfusing the corrected cells .…”

mentioning

confidence: 99%

“…Researchers have used CRISPR-based editing to correct the genetic basis of many diseases in isolated cells or animal models. [20][21][22][23][24][25][26][27][28][29] The first wave of clinical trials using CRISPR enzymes to treat inherited disorders in humans involve removing a patient's cells, editing ex vivo, and reinfusing the corrected cells. 30 Such ex vivo genome editing is currently the most technically feasible approach, and has the potential to treat devastating blood disorders like sickle cell disease and β-thalassemia.…”

mentioning

confidence: 99%

Clinical applications of CRISPR‐based genome editing and diagnostics

2019

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Clustered regularly interspaced short palindromic repeats (CRISPR)‐driven genome editing has rapidly transformed preclinical biomedical research by eliminating the underlying genetic basis of many diseases in model systems and facilitating the study of disease etiology. Translation to the clinic is under way, with announced or impending clinical trials utilizing ex vivo strategies for anticancer immunotherapy or correction of hemoglobinopathies. These exciting applications represent just a fraction of what is theoretically possible for this emerging technology, but many technical hurdles must be overcome before CRISPR‐based genome editing technology can reach its full potential. One exciting recent development is the use of CRISPR systems for diagnostic detection of genetic sequences associated with pathogens or cancer. We review the biologic origins and functional mechanism of CRISPR systems and highlight several current and future clinical applications of genome editing.

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Molecular Biology, Genetics, and Translational Models of Human Cancer

Traub¹,

Scheufele²,

Viswanathan³

et al. 2022

Holland‐Frei Cancer Medicine

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Overview Cancer is a disease of abnormal cellular behavior and characterized by molecular aberrations that promote cellular proliferation and survival. Neoplastic progression ultimately produces tumor masses that compromise local organ function, and the invasion and metastasis of malignant cells away from their tissue of origin and spread throughout the body cause a diverse array of pathophysiologic sequelae that compromise the quality and length of life of patients. A background knowledge of cancer pathogenesis and cancer models should help oncologists understand the developing approaches for optimal patient care, including the new concept of personalized medicine. This article is a basic, “methods‐oriented” survey of molecular biology directed toward the clinician or trainee who wants a fundamental understanding of this discipline. It describes the principles that underlie the procedures used most commonly by molecular biologists and provides examples of clinically relevant situations that draw on particular techniques. Molecular biology already plays an important role in clinical cancer medicine, both in terms of diagnosis (e.g., in the analysis of tumors for prognostic or pathogenetic information) and in treatment (e.g., in the production of pharmacologic and biologic agents, such as recombinant growth factors and monoclonal antibodies). We will begin with an overview of genes, gene expression, and gene cloning. Our discussion of techniques will follow the flow of genetic information as we explain the procedures used to analyze gene expression at the levels of DNA, ribonucleic acid ( RNA ), and protein. We conclude with a discussion of current cancer modeling systems in vitro and in vivo . Good general overviews of these topics can be found in several books. 1–3

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Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Cited by 287 publications

References 54 publications

Delivery of CRISPR/Cas9 for therapeutic genome editing

Delivery of CRISPR/Cas9 for therapeutic genome editing

Clinical applications of CRISPR‐based genome editing and diagnostics

Molecular Biology, Genetics, and Translational Models of Human Cancer

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