scholarly journals Gene Therapy for Pancreatic Diseases: Current Status

2018 ◽  
Vol 19 (11) ◽  
pp. 3415 ◽  
Author(s):  
Kenya Kamimura ◽  
Takeshi Yokoo ◽  
Shuji Terai
Keyword(s):  
Pancreatic Cancer ◽  
The Body ◽  
Therapeutic Options ◽  
Current Status ◽  
Endocrine Tumors ◽  
Therapeutic Approaches ◽  
Islet Cell Tumors ◽  
Pancreatic Diseases ◽  
Gene Therapies ◽  
New Strategies

The pancreas is a key organ involved in digestion and endocrine functions in the body. The major diseases of the pancreas include pancreatitis, pancreatic cancer, cystic diseases, pancreatic divisum, islet cell tumors, endocrine tumors, diabetes mellitus, and pancreatic pain induced by these diseases. While various therapeutic methodologies have been established to date, however, the improvement of conventional treatments and establishment of novel therapies are essential to improve the efficacy. For example, conventional therapeutic options, including chemotherapy, are not effective against pancreatic cancer, and despite improvements in the last decade, the mortality rate has not declined and is estimated to become the second cause of cancer-related deaths by 2030. Therefore, continuous efforts focus on the development of novel therapeutic options. In this review, we will summarize the progress toward the development of gene therapies for pancreatic diseases, with an emphasis on recent preclinical studies and clinical trials. We aim to identify new areas for improvement of the current methodologies and new strategies that will lead to safe and effective gene therapeutic approaches in pancreatic diseases.

scholarly journals Gene Therapy for Liver Cancers: Current Status from Basic to Clinics

Cancers ◽  
2019 ◽  
Vol 11 (12) ◽  
pp. 1865 ◽  
Author(s):  
Kenya Kamimura ◽  
Takeshi Yokoo ◽  
Hiroyuki Abe ◽  
Shuji Terai
Keyword(s):  
Gene Therapy ◽  
Liver Cancer ◽  
Liver Diseases ◽  
Malignant Tumors ◽  
Therapeutic Options ◽  
Endocrine Function ◽  
Current Status ◽  
Congenital Disease ◽  
Gene Therapies ◽  
Liver Cancers

The liver is a key organ for metabolism, protein synthesis, detoxification, and endocrine function, and among liver diseases, including hepatitis, cirrhosis, malignant tumors, and congenital disease, liver cancer is one of the leading causes of cancer-related deaths worldwide. Conventional therapeutic options such as embolization and chemotherapy are not effective against advanced-stage liver cancer; therefore, continuous efforts focus on the development of novel therapeutic options, including molecular targeted agents and gene therapy. In this review, we will summarize the progress toward the development of gene therapies for liver cancer, with an emphasis on recent clinical trials and preclinical studies.

Current Controversies in the Stage-Specific Multidisciplinary Management of Pancreatic Cancer

2014 ◽  
pp. e157-e164 ◽  
Author(s):  
Matthew H. G. Katz ◽  
Jerome Landry ◽  
Hedy Lee Kindler
Keyword(s):  
Pancreatic Cancer ◽  
Systemic Chemotherapy ◽  
Metastatic Cancer ◽  
Clinical Staging ◽  
Newly Diagnosed ◽  
Therapeutic Approaches ◽  
Treatment Regimens ◽  
Localized Disease ◽  
New Strategies

Encouraging therapeutic approaches and treatment regimens for patients with both localized and metastatic pancreatic cancer have emerged over the last 5 years. However, these new strategies have brought important challenges and controversy. Clinical staging criteria are constantly evolving. No system has been uniformly adopted, limiting our understanding of the role of both pancreatectomy and neoadjuvant therapies for localized disease. The role of radiation therapy for the treatment of both resectable and unresectable pancreatic cancer remains unclear despite multiple prospective studies. Although two new systemic chemotherapy regimens have essentially transformed the care of many patients with metastatic cancer, criteria to guide their use in the general population have yet to be clearly established. Herein we provide an overview of these important controversies in the context of a broad update on the stage-specific management of patients with newly diagnosed pancreatic cancer.

scholarly journals Roles of Commensal Microbiota in Pancreas Homeostasis and Pancreatic Pathologies

2015 ◽  
Vol 2015 ◽  
pp. 1-20 ◽  
Author(s):  
Camila Leal-Lopes ◽  
Fernando J. Velloso ◽  
Julia C. Campopiano ◽  
Mari C. Sogayar ◽  
Ricardo G. Correa
Keyword(s):  
Diabetes Mellitus ◽  
Human Physiology ◽  
The Body ◽  
The Other ◽  
Therapeutic Approaches ◽  
Pancreatic Diseases ◽  
Commensal Microbiota ◽  
Pathological Conditions ◽  
Metabolic Imbalance ◽  
Underlying Mechanisms

The pancreas plays a central role in metabolism, allowing ingested food to be converted and used as fuel by the cells throughout the body. On the other hand, the pancreas may be affected by devastating diseases, such as pancreatitis, pancreatic adenocarcinoma (PAC), and diabetes mellitus (DM), which generally results in a wide metabolic imbalance. The causes for the development and progression of these diseases are still controversial; therefore it is essential to better understand the underlying mechanisms which compromise the pancreatic homeostasis. The interest in the study of the commensal microbiome increased extensively in recent years, when many discoveries have illustrated its central role in both human physiology and maintenance of homeostasis. Further understanding of the involvement of the microbiome during the development of pathological conditions is critical for the improvement of new diagnostic and therapeutic approaches. In the present review, we discuss recent findings on the behavior and functions played by the microbiota in major pancreatic diseases and provide further insights into its potential roles in the maintenance of pancreatic steady-state activities.

scholarly journals Current Status of Pediatric Formulations for Chronic and Acute Children’ Diseases: Applications and Future Perspectives

2021 ◽  
Author(s):  
Panoraia Siafaka ◽  
Esra İpekçi ◽  
Emre Şefik Çağlar ◽  
Neslihan Üstündağ Okur ◽  
Derya Büyükkayhan
Keyword(s):  
Pediatric Patients ◽  
Pharmaceutical Products ◽  
Dosage Forms ◽  
The Body ◽  
Current Status ◽  
Therapeutic Approaches ◽  
Solid Dosage ◽  
Age Variations ◽  
Rare Illnesses ◽  
Taste And Smell

Infants and other children can be affected by various acute, chronic and many of them rare illnesses. Developing drugs for children is very challenging since they cannot intake tablets or hard oral solid dosage forms. Besides, most of the prescribed pediatric medications are unlicensed. The biggest issue that clinicians have to solve is that dosing in children is not based on weight or surface area of the body, as it happened in adults but is related to age variations in drug absorption, distribution, metabolism, and elimination. Thus, for pediatric patients, various therapeutic approaches have been proposed so as to develop suitable formulations such as liquid dosage forms, flexible capsules, milk-based products, etc. In addition, the administration of current pharmaceutical products to children might lead to some serious side effects which can also happen in adults but with a lower risk. Especially, infants are at high risk of getting poisoned by taking drugs used for adults. Moreover, children are very sensitive to the taste and smell of some pharmaceutical vehicles and can resist to intake them and this situation leads parents to search for tasteless and odorless medications. In this study, the current formulations for various diseases intended to be used in pediatric patients as well as various chronic and acute diseases of childhood are summarized. Authors believe that this review can help professionals who want to work with pediatric formulations to design more efficient and child-friendly drug delivery systems.

Novel Approaches for Oral Delivery of Insulin and Current Status of Oral Insulin Products

2010 ◽  
Vol 3 (3) ◽  
pp. 1057-1064 ◽  
Author(s):  
Kinesh V P ◽  
Neelam D P ◽  
Punit B ◽  
Bhavesh S.B ◽  
Pragna K. S
Keyword(s):  
Diabetes Mellitus ◽  
Oral Delivery ◽  
Proteolytic Enzymes ◽  
Oral Route ◽  
The Body ◽  
Current Status ◽  
Intestinal Lumen ◽  
Insulin Administration ◽  
Novel Approaches ◽  
Dose Dependent

Diabetes mellitus is a serious pathologic condition that is responsible for major healthcare problems worldwide and costing billions of dollars annually. Insulin replacement therapy has been used in the clinical management of diabetes mellitus for more than 84 years. The present mode of insulin administration is by the subcutaneous route through which insulin is presented to the body in a non-physiological manner having many challenges. Hence novel approaches for insulin delivery are being explored. Challenges to oral route of insulin administration are: rapid enzymatic degradation in the stomach, inactivation and digestion by proteolytic enzymes in the intestinal lumen and poor permeability across intestinal epithelium because of its high molecular weight and lack of lipophilicity. Liposomes, microemulsions, nanocubicles, and so forth have been prepared for the oral delivery of insulin. Chitosan-coated microparticles protected insulin from the gastric environment of the body and released intestinal pH. Limitations to the delivery of insulin have not resulted in fruitful results to date and there is still a need to prepare newer delivery systems, which can produce dose-dependent and reproducible effects, in addition to increased bioavailability.

Healthy Gut, Healthy Brain: The Gut Microbiome in Neurodegenerative Disorders

2020 ◽  
Vol 20 (13) ◽  
pp. 1142-1153 ◽  
Author(s):  
Sreyashi Chandra ◽  
Md. Tanjim Alam ◽  
Jhilik Dey ◽  
Baby C. Pulikkaparambil Sasidharan ◽  
Upasana Ray ◽  
...  
Keyword(s):  
Gut Microbiota ◽  
Neurodegenerative Disorders ◽  
The Body ◽  
Therapeutic Approaches ◽  
Cns Disorders ◽  
Physiological Conditions ◽  
Pathological Conditions ◽  
The Central Nervous System ◽  
Present Understanding ◽  
Lateral Sclerosis

Background: The central nervous system (CNS) known to regulate the physiological conditions of human body, also itself gets dynamically regulated by both the physiological as well as pathological conditions of the body. These conditions get changed quite often, and often involve changes introduced into the gut microbiota which, as studies are revealing, directly modulate the CNS via a crosstalk. This cross-talk between the gut microbiota and CNS, i.e., the gut-brain axis (GBA), plays a major role in the pathogenesis of many neurodegenerative disorders such as Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS) and Huntington’s disease (HD). Objective: We aim to discuss how gut microbiota, through GBA, regulate neurodegenerative disorders such as PD, AD, ALS, MS and HD. Methods: In this review, we have discussed the present understanding of the role played by the gut microbiota in neurodegenerative disorders and emphasized the probable therapeutic approaches being explored to treat them. Results: In the first part, we introduce the GBA and its relevance, followed by the changes occurring in the GBA during neurodegenerative disorders and then further discuss its role in the pathogenesis of these diseases. Finally, we discuss its applications in possible therapeutics of these diseases and the current research improvements being made to better investigate this interaction. Conclusion: We concluded that alterations in the intestinal microbiota modulate various activities that could potentially lead to CNS disorders through interactions via the GBA.

A Potential Role of Coenzyme Q10 Deficiency in Severe SARS-CoV2 Infection

2020 ◽  
Vol 5 (4) ◽  
Author(s):  
Vasilios M. Polymeropoulos ◽  
Keyword(s):  
Viral Entry ◽  
Coenzyme Q10 ◽  
Severe Infection ◽  
The Body ◽  
Therapeutic Options ◽  
Severe Illness ◽  
Infected People ◽  
Inflammatory State ◽  
Exogenous Compounds ◽  
Obesity Hypertension

There is a dramatic need for extensive research into the predictors of severe infection with SARS-CoV2 and therapeutic options for infected people. People who suffer from severe illness and higher mortality display a pattern of having specific co-morbidities (diabetes, obesity, hypertension) and are of higher age. Recent research has described methods of viral entry via receptors (ACE2, TMPRSS2) and the hyper-inflammatory state often associated with severe illness (increase in interleukins, increase in TNF-alpha). These discoveries have led to the research of currently available and developing therapies, that are helpful to patients. Deficiencies of specific vitamins and other endogenous molecules of the body should be examined to understand if a pattern exists among the people most severely affected. Coenzyme Q10 (CoQ10) is a fat-soluble substance ubiquitously expressed throughout the body that is important for the generation of ATP and mediation of inflammatory disease. CoQ10 faces a decline with increasing age, genetic predispositions, and ingestion of exogenous compounds that could reduce the level of CoQ10. Deficiencies and subsequent supplementation with CoQ10 recently has displayed encouraging results for the improvement of a wide variety of diseases. This manuscript is significant as it points to a potential relationship of CoQ10 and the population suffering from severe illness of COVID-19, and further encourages the need for research into measuring the levels of CoQ10 and vitamins to understand if levels predict severe illness and mortality. This could offer new avenues into research in combating this pandemic and potentially future therapeutic options.

scholarly journals New Aspects of the Epigenetics of Pancreatic Carcinogenesis

Epigenomes ◽  
2020 ◽  
Vol 4 (3) ◽  
pp. 18
Author(s):  
Murat Toruner ◽  
Martin E. Fernandez-Zapico ◽  
Christopher L. Pin
Keyword(s):  
Pancreatic Cancer ◽  
Dna Methylation ◽  
Survival Rate ◽  
Epigenetic Mechanisms ◽  
Therapeutic Approaches ◽  
Cancer Epigenetics ◽  
Pancreatic Carcinogenesis ◽  
New Therapeutic Approaches ◽  
Environmental Events ◽  
Underlying Pathogenesis

Pancreatic cancer remains among the deadliest forms of cancer with a 5 year survival rate less than 10%. With increasing numbers being observed, there is an urgent need to elucidate the pathogenesis of pancreatic cancer. While both contribute to disease progression, neither genetic nor environmental factors completely explain susceptibility or pathogenesis. Defining the links between genetic and environmental events represents an opportunity to understand the pathogenesis of pancreatic cancer. Epigenetics, the study of mitotically heritable changes in genome function without a change in nucleotide sequence, is an emerging field of research in pancreatic cancer. The main epigenetic mechanisms include DNA methylation, histone modifications and RNA interference, all of which are altered by changes to the environment. Epigenetic mechanisms are being investigated to clarify the underlying pathogenesis of pancreatic cancer including an increasing number of studies examining the role as possible diagnostic and prognostic biomarkers. These mechanisms also provide targets for promising new therapeutic approaches for this devastating malignancy.

scholarly journals Disease Mechanisms and Therapeutic Approaches in C9orf72 ALS-FTD

Biomedicines ◽  
2021 ◽  
Vol 9 (6) ◽  
pp. 601
Author(s):  
Keith Mayl ◽  
Christopher E. Shaw ◽  
Youn-Bok Lee
Keyword(s):  
The Body ◽  
The Novel ◽  
Therapeutic Approaches ◽  
Gain Of Function ◽  
Pathogenic Mechanisms ◽  
Hexanucleotide Repeat ◽  
Primary Driver ◽  
Hexanucleotide Repeat Expansion ◽  
Expansion Mutation ◽  
Lateral Sclerosis

A hexanucleotide repeat expansion mutation in the first intron of C9orf72 is the most common known genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia. Since the discovery in 2011, numerous pathogenic mechanisms, including both loss and gain of function, have been proposed. The body of work overall suggests that toxic gain of function arising from bidirectionally transcribed repeat RNA is likely to be the primary driver of disease. In this review, we outline the key pathogenic mechanisms that have been proposed to date and discuss some of the novel therapeutic approaches currently in development.

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