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The New England Journal of Medicine Jun 2023
Review
Topics: Humans; Artificial Intelligence; Molecular Medicine
PubMed: 37379136
DOI: 10.1056/NEJMra2204787 -
Med (New York, N.Y.) Feb 2021Studies of the major hemoglobin disorders, β-thalassemia and sickle cell disease (SCD), have laid a foundation for molecular medicine. While enormous progress has been... (Review)
Review
Studies of the major hemoglobin disorders, β-thalassemia and sickle cell disease (SCD), have laid a foundation for molecular medicine. While enormous progress has been made in understanding gene structure and regulation, translating molecular insights to therapy for the many individuals affected with these disorders has been challenging. Advances in three activities have recently converged to bring novel genetic and potentially curative treatments to clinical trials. First, improved lentiviral vectors for gene transfer into hematopoietic stem cells have revived somatic gene therapy for blood disorders. Second, elucidation of regulatory factors and mechanisms that control the normal developmental switch from fetal to adult hemoglobin has provided a route to reactivation of the fetal form for therapy. Third, revolutionary methods of gene engineering permit molecular insights to be leveraged for patients. Here I review how the promise of molecular medicine to bring transformative treatments to the clinical arena is finally being realized.
Topics: Adult; Genetic Therapy; Hemoglobinopathies; Hemoglobins; Humans; Molecular Medicine; beta-Thalassemia
PubMed: 33688634
DOI: 10.1016/j.medj.2020.12.011 -
Dermatologic Clinics Jan 2023Melanoma is the most lethal form of skin cancer although surgery is often curative when combined with early screening and prevention. In recurrent or advanced cancer,... (Review)
Review
Melanoma is the most lethal form of skin cancer although surgery is often curative when combined with early screening and prevention. In recurrent or advanced cancer, the emergence of chemotherapy, targeted therapy, and immune checkpoint inhibitors has demonstrated promising clinical outcomes. Such approaches can remarkably halt the progression of disease for many years, although are limited by the acquisition of resistance. The development and approval of combination therapies has further changed the treatment paradigm for certain melanomas. This review focuses on the current state of melanoma epidemiology and recent advancements in melanoma screening, histopathological classification, staged excision (i.e. wide local excision, sentinel lymph node biopsy, and Mohs micrographic surgery), and systemic treatment.
Topics: Humans; Molecular Medicine; Melanoma; Skin Neoplasms; Sentinel Lymph Node Biopsy; Mohs Surgery
PubMed: 36410983
DOI: 10.1016/j.det.2022.07.017 -
EMBO Molecular Medicine Nov 2020Sarcomas are heterogeneous and clinically challenging soft tissue and bone cancers. Although constituting only 1% of all human malignancies, sarcomas represent the... (Review)
Review
Sarcomas are heterogeneous and clinically challenging soft tissue and bone cancers. Although constituting only 1% of all human malignancies, sarcomas represent the second most common type of solid tumors in children and adolescents and comprise an important group of secondary malignancies. More than 100 histological subtypes have been characterized to date, and many more are being discovered due to molecular profiling. Owing to their mostly aggressive biological behavior, relative rarity, and occurrence at virtually every anatomical site, many sarcoma subtypes are in particular difficult-to-treat categories. Current multimodal treatment concepts combine surgery, polychemotherapy (with/without local hyperthermia), irradiation, immunotherapy, and/or targeted therapeutics. Recent scientific advancements have enabled a more precise molecular characterization of sarcoma subtypes and revealed novel therapeutic targets and prognostic/predictive biomarkers. This review aims at providing a comprehensive overview of the latest advances in the molecular biology of sarcomas and their effects on clinical oncology; it is meant for a broad readership ranging from novices to experts in the field of sarcoma.
Topics: Adolescent; Bone Neoplasms; Child; Humans; Molecular Medicine; Osteosarcoma; Sarcoma; Soft Tissue Neoplasms
PubMed: 33047515
DOI: 10.15252/emmm.201911131 -
Trends in Molecular Medicine Sep 2020
Topics: Humans; Molecular Medicine
PubMed: 32857967
DOI: 10.1016/j.molmed.2020.07.003 -
Neuro-oncology Aug 2021The fifth edition of the WHO Classification of Tumors of the Central Nervous System (CNS), published in 2021, is the sixth version of the international standard for the... (Review)
Review
The fifth edition of the WHO Classification of Tumors of the Central Nervous System (CNS), published in 2021, is the sixth version of the international standard for the classification of brain and spinal cord tumors. Building on the 2016 updated fourth edition and the work of the Consortium to Inform Molecular and Practical Approaches to CNS Tumor Taxonomy, the 2021 fifth edition introduces major changes that advance the role of molecular diagnostics in CNS tumor classification. At the same time, it remains wedded to other established approaches to tumor diagnosis such as histology and immunohistochemistry. In doing so, the fifth edition establishes some different approaches to both CNS tumor nomenclature and grading and it emphasizes the importance of integrated diagnoses and layered reports. New tumor types and subtypes are introduced, some based on novel diagnostic technologies such as DNA methylome profiling. The present review summarizes the major general changes in the 2021 fifth edition classification and the specific changes in each taxonomic category. It is hoped that this summary provides an overview to facilitate more in-depth exploration of the entire fifth edition of the WHO Classification of Tumors of the Central Nervous System.
Topics: Brain; Central Nervous System; Central Nervous System Neoplasms; Humans; Pathology, Molecular; World Health Organization
PubMed: 34185076
DOI: 10.1093/neuonc/noab106 -
Biophysical Chemistry 2016Molecular medicine is founded on the synergy between Chemistry, Physics, Biology and Medicine, with the ambitious goal of tackling diseases from a molecular perspective.... (Review)
Review
Molecular medicine is founded on the synergy between Chemistry, Physics, Biology and Medicine, with the ambitious goal of tackling diseases from a molecular perspective. This Review aims at retracing a personal outlook of the birth and development of molecular medicine, as well as at highlighting some of the most urgent challenges linked to aging and represented by incurable neurodegenerative diseases caused by protein misfolding. Furthermore, we emphasize the emerging role of the retromer dysfunctions and improper protein sorting in Alzheimer's disease and other important neurological disordered.
Topics: Aging; Humans; Molecular Medicine; Neurodegenerative Diseases; Protein Folding; Protein Transport; Vesicular Transport Proteins
PubMed: 27214761
DOI: 10.1016/j.bpc.2016.05.004 -
EMBO Molecular Medicine Apr 2017Incoming Chief Editor Philippe Sansonetti shares his vision of the role of the journal in the burgeoning areas of molecular medicine.
Incoming Chief Editor Philippe Sansonetti shares his vision of the role of the journal in the burgeoning areas of molecular medicine.
Topics: Editorial Policies; Humans; Molecular Diagnostic Techniques; Molecular Medicine; Molecular Targeted Therapy; Periodicals as Topic
PubMed: 28314782
DOI: 10.15252/emmm.201707746 -
Trends in Molecular Medicine Apr 2021
Topics: Biographies as Topic; Humans; Molecular Medicine
PubMed: 33685849
DOI: 10.1016/j.molmed.2021.02.002 -
Chemical Society Reviews Dec 2015
Topics: Antineoplastic Agents; Humans; Molecular Medicine; Neoplasms
PubMed: 26595319
DOI: 10.1039/c5cs90123e