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Advancements in Personalized Medicine: Integrating Genomic Insights and Therapeutic Strategies

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Genetics and Genomics".

Deadline for manuscript submissions: 20 June 2025 | Viewed by 6819

Special Issue Editor

Special Issue Information

Dear Colleagues,

This Special Issue delves into the forefront of personalized medicine, wherein the fusion of genomic insights with cutting-edge bio-molecular features will pave the way for innovative therapeutic strategies. Emphasizing the integration of genomic data, we invite contributions that spotlight the intricate molecular mechanisms underpinning personalized treatment approaches. From explorations of biomarkers to in-depth analyses of bio-molecular pathways, this issue aims to bridge the gap between genomic discoveries and their practical application in tailoring therapeutic interventions. We welcome submissions that showcase the synergy between clinical insights and rigorous bio-molecular experiments, encouraging more comprehensive understanding of personalized medicine’s evolving landscape.

Dr. Mariarosaria Boccellino
Guest Editor

Manuscript Submission Information

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Keywords

  • personalized medicine
  • genomic insights
  • therapeutic strategies
  • molecular profiling
  • precision medicine
  • molecular targets
  • precision oncology
  • genomic biomarkers

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Published Papers (3 papers)

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Research

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19 pages, 3165 KiB  
Article
Genomic Characterization of Preclinical Prostate Cancer Cell Line Models
by Erica L. Beatson, Emily N. Risdon, Giulia C. Napoli, Douglas K. Price, Cindy H. Chau and William D. Figg
Int. J. Mol. Sci. 2024, 25(11), 6111; https://doi.org/10.3390/ijms25116111 - 1 Jun 2024
Viewed by 2053
Abstract
As we move into the era of precision medicine, the growing relevance of genetic alterations to prostate cancer (PCa) development and treatment demonstrates the importance of characterizing preclinical models at the genomic level. Our study investigated the genomic characterization of eight PCa cell [...] Read more.
As we move into the era of precision medicine, the growing relevance of genetic alterations to prostate cancer (PCa) development and treatment demonstrates the importance of characterizing preclinical models at the genomic level. Our study investigated the genomic characterization of eight PCa cell lines to understand which models are clinically relevant. We designed a custom AmpliSeq DNA gene panel that encompassed key molecular pathways targeting AR signaling, apoptosis, DNA damage repair, and PI3K/AKT/PTEN, in addition to tumor suppressor genes. We examined the relationship between cell line genomic alterations and therapeutic response. In addition, using DepMap’s Celligner tool, we identified which preclinical models are most representative of specific prostate cancer patient populations on cBioPortal. These data will help investigators understand the genetic differences in preclinical models of PCa and determine which ones are relevant for use in their translational research. Full article
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Review

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19 pages, 915 KiB  
Review
Gene Therapy for Inherited Liver Disease: To Add or to Edit
by Yue Chen, Niek P. van Til and Piter J. Bosma
Int. J. Mol. Sci. 2024, 25(23), 12514; https://doi.org/10.3390/ijms252312514 - 21 Nov 2024
Cited by 1 | Viewed by 1337
Abstract
Patients suffering from an inherited severe liver disorder require lifelong treatment to prevent premature death. Until recently, the only curative treatment option was liver transplantation, which requires lifelong immune suppression. Now, liver-directed gene therapy, which is a much less invasive procedure, has become [...] Read more.
Patients suffering from an inherited severe liver disorder require lifelong treatment to prevent premature death. Until recently, the only curative treatment option was liver transplantation, which requires lifelong immune suppression. Now, liver-directed gene therapy, which is a much less invasive procedure, has become a market-approved treatment for hemophilia A and B. This may pave the way for it to become the treatment of choice for many other recessive inherited liver disorders with loss-of-function mutations. Inherited liver disease with toxic-gain-of-function or intrinsic hepatocyte damage may require alternative applications, such as integrating vectors or genome editing technologies, that can provide permanent or specific modification of the genome. We present an overview of currently available gene therapy strategies, i.e., gene supplementation, gene editing, and gene repair investigated in preclinical and clinical studies to treat inherited severe liver disorders. The advantages and limitations of these gene therapy applications are discussed in relation to the underlying disease mechanism. Full article
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13 pages, 1821 KiB  
Review
Epigenetics, Microbiome and Personalized Medicine: Focus on Kidney Disease
by Giuseppe Gigliotti, Rashmi Joshi, Anam Khalid, David Widmer, Mariarosaria Boccellino and Davide Viggiano
Int. J. Mol. Sci. 2024, 25(16), 8592; https://doi.org/10.3390/ijms25168592 - 6 Aug 2024
Cited by 3 | Viewed by 2449
Abstract
Personalized medicine, which involves modifying treatment strategies/drug dosages based on massive laboratory/imaging data, faces large statistical and study design problems. The authors believe that the use of continuous multidimensional data, such as those regarding gut microbiota, or binary multidimensional systems properly transformed into [...] Read more.
Personalized medicine, which involves modifying treatment strategies/drug dosages based on massive laboratory/imaging data, faces large statistical and study design problems. The authors believe that the use of continuous multidimensional data, such as those regarding gut microbiota, or binary multidimensional systems properly transformed into a continuous variable, such as the epigenetic clock, offer an advantageous scenario for the design of trials of personalized medicine. We will discuss examples focusing on kidney diseases, specifically on IgA nephropathy. While gut dysbiosis can provide a treatment strategy to restore the standard gut microbiota using probiotics, transforming epigenetic omics data into epigenetic clocks offers a promising tool for personalized acute and chronic kidney disease care. Epigenetic clocks involve a complex transformation of DNA methylome data into estimated biological age. These clocks can identify people at high risk of developing kidney problems even before symptoms appear. Some of the effects of both the epigenetic clock and microbiota on kidney diseases seem to be mediated by endothelial dysfunction. These “big data” (epigenetic clocks and microbiota) can help tailor treatment plans by pinpointing patients likely to experience rapid declines or those who might not need overly aggressive therapies. Full article
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