Target Discovery in Precision Medicine

A special issue of Journal of Personalized Medicine (ISSN 2075-4426). This special issue belongs to the section "Clinical Medicine, Cell, and Organism Physiology".

Deadline for manuscript submissions: closed (10 July 2022) | Viewed by 8247

Special Issue Editor


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Guest Editor
St Jude Children's Research Hospital, Memphis, TN, USA
Interests: cancer biology; regulation of gene expression; big data; target identification; co-regulation; high-throughput screening; pipeline development; computational tools; molecular dynamics simulations; machine learning

Special Issue Information

Dear Colleagues,

Precision medicine takes advantage of patient variabilities to produce tailored, individualized therapies. These variabilities come from differences in DNA sequences, mRNA expression, and protein expression, among other things. Ultimately, the goal is to understand these differences in relation to one or more targets of interest. Once a target is identified as selectively responsive in a certain patient or cohort, it can be exploited for precision therapy. The discovery of disease-specific targets is therefore critical and of great interest to the field of precision medicine.

This Special Issue aims to highlight current trends in target discovery research as well as their clinical applications in diseases related to precision medicine. Targets are defined broadly and can include things such as single-nucleotide polymorphisms (SNPs), RNA/protein expression, and metabolites.

Recently, more advanced techniques in target discovery have emerged. These techniques employ a higher throughput than was previously achievable, including greater sample sizes and types of data. Some of these include the following: high-throughput DNA sequencing to enable detection of genetic mutations, RNA sequencing to allow transcriptome-wide expression profiling, high-throughput drug screening to help elucidate disease-specific vulnerabilities, and genome-wide CRISPR screens to identify actionable dependencies of a given disease. In addition to these experimental techniques, computational approaches take advantage of one or more of these big datasets to develop tools capable of identifying patterns showing therapeutic potential. Collectively, these and other techniques are actively being used to unlock new targets which can be further researched for precision medicine.

We invite submissions to this Special Issue which include original research, review articles, and data analyses related to the discovery of targets which may be of use in precision medicine. We welcome articles relating to any disease.

Dr. William C. Wright
Guest Editor

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Keywords

  • drug target
  • dependency
  • target discovery
  • therapeutics
  • sequencing
  • screening

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Published Papers (2 papers)

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Research

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19 pages, 612 KiB  
Article
Aspects of Tertiary Prevention in Patients with Primary Open Angle Glaucoma
by Gabriel Zeno Munteanu, Zeno Virgiliu Ioan Munteanu, George Roiu, Cristian Marius Daina, Raluca Moraru, Liviu Moraru, Cristian Trambitas, Dana Badau and Lucia Georgeta Daina
J. Pers. Med. 2021, 11(9), 830; https://doi.org/10.3390/jpm11090830 - 24 Aug 2021
Cited by 12 | Viewed by 2372
Abstract
The purpose of the study is to assess the health of patients in the activity of tertiary prevention dedicated to preventing blindness caused by POAG (primary glaucoma with open angle and high tension) and NTG (primary glaucoma with open-angle and statistically normal tension—particular [...] Read more.
The purpose of the study is to assess the health of patients in the activity of tertiary prevention dedicated to preventing blindness caused by POAG (primary glaucoma with open angle and high tension) and NTG (primary glaucoma with open-angle and statistically normal tension—particular form of glaucoma with open angle) and preservation of the remaining visual function. The design of the study is epidemiological, observational, descriptive and retrospective, and uses only the data recorded in the existing records in the archives of the Ophthalmology office within the Integrated Outpatient Clinic of the Emergency Clinical Hospital of Oradea (IOCECHO) during the years 1999–2019 (anamnestic data; objective examination and paraclinical examination: intraocular pressure—IOP and visual field—VF). The methods of the study included the standardized protocol: anamnesis, physical ophthalmological examination, IOP determination, and computerized perimetry with the “Fast Threshold” strategy performed with the “Opto AP-300” perimeter. The obtained results were statistically processed with a specialized software (S.P.S.S.—I.B.M. Statistics version 22). The study examined the available data of 522 patients of which 140 were men (26.8%) and 382 were women (73.2%). The gender ratio was 0.37. In the period 1999–2019, 150,844 people with ophthalmic pathology were consulted in the Ophthalmology office of IOCECHO out of which 522 patients (0.35%) were diagnosed with primitive open-angle glaucoma, 184 people (35.2%) presented high IOP (POAG), and 338 people (64.8%) had statistically normal IOP (NTG). The annual proportion of cases diagnosed with glaucoma in the total number of patients examined was between 0.1% (2005; 2008; 2010) and 2.4% in 2012, when 101 people were detected. In the studied records, no cases of uni- and/or bilateral blindness were mentioned. The mean age of glaucoma patients at the first consultation was 60.81 ± 12.14 years with high frequencies in the 55–69 age groups and at the last consultation it was 66.10 ± 12.47 years with high frequencies in the age groups between 60–74 years. Monitoring and treatment of glaucoma patients was beneficial; IOP decreased statistically significantly: in patients with POAG by 46.16%, from 30.50 ± 7.98 mmHg to 16.42 ± 3.01 mmHg (p = 0.000) and in those with NTG by 17.44%, at 16.39 ± 3.66 mmHg at 13.53 ± 1.92 mmHG (p = 0.000). The duration of treatment and monitoring was on average 5.1 ± 3.4 years, for 184 patients (35.2%) with POAG and 5.1 ± 3.8 years for 338 patients (64.8%) with NTG. Tertiary prevention of glaucoma, by providing specialized care, ensures effective control of IOP and implicitly of the long-term evolution of the disease. IOP is the only modifiable risk factor in patients with POAG and NTG and its decrease prevents the progression of the disease and emphasizes the importance of early diagnosis and treatment. The management of the glaucoma patient consisted of: complete ophthalmological examination (subjective and objective), paraclinical examination with IOP, and VF measurement (valuable ophthalmological diagnostic tool) for disease detection and effective assessment of disease progression in order to improve the process of therapeutic decision making. Full article
(This article belongs to the Special Issue Target Discovery in Precision Medicine)
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Review

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22 pages, 981 KiB  
Review
Precision Medicine for Hepatocellular Carcinoma: Clinical Perspective
by Danijel Galun, Dragana Mijac, Aleksandar Filipovic, Aleksandar Bogdanovic, Marko Zivanovic and Dragan Masulovic
J. Pers. Med. 2022, 12(2), 149; https://doi.org/10.3390/jpm12020149 - 24 Jan 2022
Cited by 16 | Viewed by 4803
Abstract
Hepatocellular carcinoma (HCC) is one of the major malignant diseases worldwide, characterized by growing incidence and high mortality rates despite apparent improvements in surveillance programs, diagnostic and treatment procedures, molecular therapies, and numerous research initiatives. Most HCCs occur in patients with liver cirrhosis, [...] Read more.
Hepatocellular carcinoma (HCC) is one of the major malignant diseases worldwide, characterized by growing incidence and high mortality rates despite apparent improvements in surveillance programs, diagnostic and treatment procedures, molecular therapies, and numerous research initiatives. Most HCCs occur in patients with liver cirrhosis, and the competing mortality risks from the tumor and the cirrhosis should be considered. Presently, previously identified risk factors, such as hepatitis virus infection, hepatic inflammation and fibrosis, and metabolic syndrome, may be used as chemoprevention targets. The application of precision medicine for HCC management challenges the one-size-fits-all concept; moreover, patients should no longer be treated entirely according to the histology of their tumor but based on molecular targets specific to their tumor biology. Next-generation sequencing emphasizes HCC molecular heterogeneity and aids our comprehension of possible vulnerabilities that can be exploited. Moreover, genetic sequencing as part of a precision medicine concept may work as a promising tool for postoperative cancer monitoring. The use of genetic and epigenetic markers to identify therapeutic vulnerability could change the diagnosis and treatment of HCC, which so far was based on Barcelona clinic liver cancer (BCLC) staging. In daily clinical practice, the shift from a stage-oriented to a therapeutic-oriented approach is needed to direct the choice of HCC treatment toward the potentially most effective option on an individual basis. The important factor in precision medicine is the promotion of patient management based on the individual approach, knowing that the final decision must be approved by a multidisciplinary expert team. Full article
(This article belongs to the Special Issue Target Discovery in Precision Medicine)
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