Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

Khoshandam, Mohadeseh; Soltaninejad, Hossein; Mousazadeh, Marziyeh; Hamidieh, Amir Ali; Hosseinkhani, Saman

doi:10.1016/j.gendis.2023.02.027

Cited by 32 publications

(12 citation statements)

References 153 publications

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“…However, this phenomenon can be inhibited by the STING pathway inhibitor amlexanox [ 43 ]. Clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated nuclease 9 (Cas9), a simple and easy-to-operate gene editing technology that has a wide range of applications in targeting and preventing severe diseases, including CVDs, has been one of the greatest advances in biomedicine in the last two decades [ 130 ]. Zhao et al [ 131 ] revealed that CRISPR-Cas9 gene editing delivered by an adeno-associated virus ameliorates familial hypercholesterolemia due to low-density lipoprotein receptor (LDLR) mutations, reduces the aortic AS plaque area, and attenuates inflammatory infiltration.…”

Section: Future Expectationsmentioning

confidence: 99%

The cGAS-STING pathway in cardiovascular diseases: from basic research to clinical perspectives

An,

Li,

Chen

et al. 2024

Cell Biosci

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The cyclic guanosine monophosphate (GMP)-adenosine monophosphate (AMP) synthase-stimulator of interferon genes (cGAS-STING) signaling pathway, an important component of the innate immune system, is involved in the development of several diseases. Ectopic DNA-induced inflammatory responses are involved in several pathological processes. Repeated damage to tissues and metabolic organelles releases a large number of damage-associated molecular patterns (mitochondrial DNA, nuclear DNA, and exogenous DNA). The DNA fragments released into the cytoplasm are sensed by the sensor cGAS to initiate immune responses through the bridging protein STING. Many recent studies have revealed a regulatory role of the cGAS-STING signaling pathway in cardiovascular diseases (CVDs) such as myocardial infarction, heart failure, atherosclerosis, and aortic dissection/aneurysm. Furthermore, increasing evidence suggests that inhibiting the cGAS-STING signaling pathway can significantly inhibit myocardial hypertrophy and inflammatory cell infiltration. Therefore, this review is intended to identify risk factors for activating the cGAS-STING pathway to reduce risks and to simultaneously further elucidate the biological function of this pathway in the cardiovascular field, as well as its potential as a therapeutic target.

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Section: Future Expectationsmentioning

confidence: 99%

The cGAS-STING pathway in cardiovascular diseases: from basic research to clinical perspectives

An,

Li,

Chen

et al. 2024

Cell Biosci

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“…Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a promising genetic approach recently emerging as a potentially effective treatment against hyperlipidemia. Compared to the other editing approaches, due to its higher specificity and efficacy, CRISPR/Cas9 has been thoroughly investigated to reduce Lp(a) levels and target the PCSK9 and LPA genes [ 90 ]. A single infusion was found to knock down PCSK9 in almost every hepatocyte of cynomolgus monkeys and lowered LDL-C by up to 60% for a period of 8 months.…”

Section: Future Perspectivesmentioning

confidence: 99%

Novel Therapeutic Approaches for the Management of Elevated Lipoprotein(a): From Traditional Agents to Future Treatment Options

Paragh,

Zilahi,

Kolozsvári

et al. 2024

Life

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Cardiovascular disease is the leading cause of mortality worldwide. Despite the availability of effective low-density lipoprotein cholesterol (LDL-C) lowering agents, an increased cardiovascular risk is still observed in individuals with therapeutic LDL-C levels. One of these cardiovascular risk factors is elevated plasma lipoprotein(a) (Lp(a)) concentration, which maintains chronic inflammation through the increased presence of oxidized phospholipids on its surface. In addition, due to its 90 percent homology with the fibrinolytic proenzyme plasminogen, Lp(a) exhibits atherothrombotic effects. These may also contribute to the increased cardiovascular risk in individuals with high Lp(a) levels that previous epidemiological studies have shown to exist independently of LDL-C and other lipid parameters. In this review, the authors overview the novel therapeutic options to achieve effective Lp(a) lowering treatment, which may help to define tailored personalized medicine and reduce the residual cardiovascular risk in high-risk patients. Agents that increase LDL receptor expression, including statins, proprotein convertase subtilisin kexin type 9 inhibitors, and LDL production inhibitors, are also discussed. Other treatment options, e.g., cholesterolester transfer protein inhibitors, nicotinic acid derivatives, thyroid hormone mimetics, lipoprotein apheresis, as well as apolipoprotein(a) reducing antisense oligonucleotides and small interfering RNAs, are also evaluated.

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“… 16 , 44 , 45 , 46 Many of these limitations can be overcome using the CRISPR-Cas9-based gene editing tool as an easy and available method. 47 Accordingly, current research efforts are focused on precise CAR T cell engineering with conventional CRISPR-Cas9 systems or novel editors that can generate the desired mutations with or without inducing a double-stranded DNA break into the genome. 48 These tools and strategies can be directly applied to target negative regulators of T cell function, insert therapeutic genes to specific loci, and generate universal CAR allogeneic T cell products for on-demand immunotherapy ( Fig.…”

Section: Crispr Gene Editing Toolmentioning

confidence: 99%

CRISPR, CAR-T, and NK: Current applications and future perspectives

Khoshandam,

Soltaninejad,

Hamidieh

et al. 2024

Genes & Diseases

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Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

Cited by 32 publications

References 153 publications

The cGAS-STING pathway in cardiovascular diseases: from basic research to clinical perspectives

The cGAS-STING pathway in cardiovascular diseases: from basic research to clinical perspectives

Novel Therapeutic Approaches for the Management of Elevated Lipoprotein(a): From Traditional Agents to Future Treatment Options

CRISPR, CAR-T, and NK: Current applications and future perspectives

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