Natural Compounds as Therapeutic Agents in the Treatment Cystic Fibrosis

Dey, Isha; Shah, Kalpit; Bradbury, Neil A.

doi:10.4172/2157-7412.1000284

Cited by 30 publications

(26 citation statements)

References 52 publications

(54 reference statements)

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“…Therefore, anti-in ammatory treatments are an inevitable therapy for CF patients. Curcumin is the main ingredient in turmeric with a substantial impact on the reduction of in ammatory factors (8) (9). Previous ndings have indicated that curcumin could correct the CFTR protein function (corrector) in the F508del mutation, which is associated with the CFTR protein misfolding (10).…”

Section: Background and Rationale {6a}mentioning

confidence: 99%

The Effects of Nano-Curcumin as A Nutritional Strategy on Clinical and Inflammatory Factors in Children with Cystic Fibrosis: A Protocol Study and Review Article

Kianifar¹,

Talebi²,

Safarian³

et al. 2020

Preprint

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Background: Cystic fibrosis(CF) is a genetic disorder, which is caused by the CFTR protein defects. Along with CFTR dysfunction, inflammation plays a key role in the disease outcomes. Inflammation may develop due to the internal dysfunction of the CFTR protein or external factors. Curcumin affects the CFTR protein function primarily as a corrector and potentiator and secondary as an anti-inflammatory and antimicrobial agent. The present study aims to assess the impact of Nano-curcumin on clinical and inflammatory markers in children with CF.Methods: This prospective, double blind control trial will be conducted at Akbar Children's Hospital in Mashhad, Iran. children with CF will be enrolled based on the eligibility criteria. Placebo and curcumin with the maximum dose of 80 milligrams considering the body surface of the patients will be administrated for three months. The primary outcome is to evaluate inflammation based on serum interleukin-6, interleukin-10, and hs-CRP, stool calprotectin, and neutrophil count of nasopharyngeal swab. The secondary outcome involved clinical assessment via spirometry, anthropometrics, and quality of life. They will be assessed before and after three mounts.Discussion: Due to the multifarious effects of curcumin on CF disease, it can be used as a nutritional strategy in the treatment of cystic fibrosis.Trial registration: Iranian Registry of Clinical Trials, IRCT20200705048018N1, Registered on 2020-07-10, https://en.irct.ir/search/result?query=IRCT20200705048018N1

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Section: Background and Rationale {6a}mentioning

confidence: 99%

The Effects of Nano-Curcumin as A Nutritional Strategy on Clinical and Inflammatory Factors in Children with Cystic Fibrosis: A Protocol Study and Review Article

Kianifar¹,

Talebi²,

Safarian³

et al. 2020

Preprint

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“…6,48 PCs hold the promise of being able to reduce the severity of several genetic diseases, but it is important to note that the number of PCs described for different targets is low. For instance, in cystic fibrosis (CF), a number of PCs and PRs that might rescue CF transmembrane conductance regulator (CFTR) have recently been identified, 34,[49][50][51] one of which is the PR roscovitine, which is involved in calcium regulation. 52 The use of therapeutic autophagy inducers has also been tested in CF.…”

Section: Pharmacological Chaperonesmentioning

confidence: 99%

Protein misfolding diseases: Prospects of pharmacological treatment

et al. 2017

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Protein misfolding has been linked to numerous inherited diseases. Loss‐ and gain‐of‐function mutations (common features of genetic diseases) may cause the destabilization of proteins, leading to alterations in their properties and/or cellular location, resulting in their incorrect functioning. Misfolded proteins can, however, be rescued via the use of proteostasis regulators and/or pharmacological chaperones, suggesting that treatments with small molecules might be developed for a range of genetic diseases. This work describes the potential of these small molecules in this respect, including for the treatment of congenital disorder of glycosylation (CDG) due to phosphomannomutase 2 deficiency (PMM2‐CDG).

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“…Curcumin has been reported to correct localization of ATP7B -R778L and to facilitate Cu efflux from patient-derived HLCs. However, in phase 1 clinical trials for CFTR correction, dietary curcumin was found to have very poor bioavailability and very low serum concentration[ 15 ]. Thus, the efficacy of this corrector for WD treatment remains questionable.…”

Section: Correction Of Dysfunctional Atp7bmentioning

confidence: 99%

Wilson’s disease: Prospective developments towards new therapies

Ranucci

Polishchuck

Iorio

2017

WJG

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Wilson’s disease (WD) is an autosomal recessive disorder of copper metabolism, caused by mutations in the ATP7B gene. A clear demand for novel WD treatment strategies has emerged. Although therapies using zinc salts and copper chelators can effectively cure WD, these drugs exhibit limitations in a substantial pool of WD patients who develop intolerance and/or severe side effects. Several lines of research have indicated intriguing potential for novel strategies and targets for development of new therapies. Here, we review these new approaches, which comprise correction of ATP7B mutants and discovery of new compounds that circumvent ATP7B-deficiency, as well as cell and gene therapies. We also discuss whether and when these new therapeutic strategies will be translated into clinical use, according to the key requirements for clinical trials that remain to be met. Finally, we discuss the hope for the current rapidly developing research on molecular mechanisms underlying WD pathogenesis and for the related potential therapeutic targets to provide a solid foundation for the next generation of WD therapies that may lead to an effective, tolerable and safe cure.

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Natural Compounds as Therapeutic Agents in the Treatment Cystic Fibrosis

Cited by 30 publications

References 52 publications

The Effects of Nano-Curcumin as A Nutritional Strategy on Clinical and Inflammatory Factors in Children with Cystic Fibrosis: A Protocol Study and Review Article

The Effects of Nano-Curcumin as A Nutritional Strategy on Clinical and Inflammatory Factors in Children with Cystic Fibrosis: A Protocol Study and Review Article

Protein misfolding diseases: Prospects of pharmacological treatment

Wilson’s disease: Prospective developments towards new therapies

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