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Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Haemophilia
January 2025
Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Introduction: Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a common and understandable language to discuss gene editing will be essential to ensure these treatments can be deployed in a safe and effective manner with fully informed and shared decision-making between healthcare professionals (HCPs) and PWH. A lexicon explaining and clarifying key concepts is one potential tool to address these aims.
View Article and Find Full Text PDFInvestigation of the Potency of KALA and REV Cell-Penetrating Peptides for In Vitro/In Vivo Delivery of an HPV Multiepitope DNA Construct.
J Pept Sci
March 2025
Department of Hepatitis and AIDS, Pasteur Institute of Iran, Tehran, Iran.
Developing human papillomavirus (HPV) therapeutic DNA vaccines requires an effective delivery system, such as cell-penetrating peptides (CPPs). In the current study, the multiepitope DNA constructs harboring the immunogenic and conserved epitopes of the L1, L2, and E7 proteins of HPV16/18 (pcDNA-L1-L2-E7 and pEGFP-L1-L2-E7) were delivered using KALA and REV CPPs with different properties in vitro and in vivo. Herein, after confirmation of the REV/DNA and KALA/DNA complexes, their stability was investigated against DNase I and serum protease.
View Article and Find Full Text PDFDeep analysis of the major histocompatibility complex genetic associations using covariate analysis and haploblocks unravels new mechanisms for the molecular etiology of Elite Control in AIDS.
BMC Immunol
January 2025
Laboratoire Génomique, Bioinformatique, et Chimie Moléculaire, Conservatoire National des Arts et Métiers, 2 rue Conté 75003, Paris, EA7528, France.
Introduction: We have reanalyzed the genomic data from the International Collaboration for the Genomics of HIV (ICGH), focusing on HIV-1 Elite Controllers (EC).
Methods: A genome-wide association study (GWAS) was performed, comparing 543 HIV-1 EC individuals with 3,272 uninfected controls (CTR) of European ancestry. 8 million single nucleotide polymorphisms (SNPs) and HLA class I and class II gene alleles were imputed to compare EC and CTR.
Non-acquired immunodeficiency syndrome defining malignancies in people living with haemophilia and human immunodeficiency virus after direct-acting antiviral era.
Glob Health Med
October 2024
Division of Infectious Diseases, Advanced Clinical Research Centre, Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
Article Synopsis
- The study investigates non-acquired immunodeficiency syndrome-defining malignancies (NADMs) among people living with hemophilia and HIV in Japan, focusing on the period after the approval of direct-acting antivirals for hepatitis C virus (HCV).
- A nationwide survey was conducted across 395 HIV treatment facilities, collecting data on 328 individuals from 2015 to 2022, which revealed that liver cancer was the most common NADM, accounting for 43% of cases identified.
- The findings indicate a significantly higher rate of liver cancer and other malignancies in this population, particularly in younger patients, emphasizing the importance of regular cancer screening and preventive strategies for PLWH
Biological Barriers for Drug Delivery and Development of Innovative Therapeutic Approaches in HIV, Pancreatic Cancer, and Hemophilia A/B.
Pharmaceutics
September 2024
WIR-Walk In Ruhr, Center for Sexual Health & Medicine, Department of Dermatology, Venerology and Allergology, Ruhr-University Bochum, 44787 Bochum, Germany.
Article Synopsis
- Biological barriers pose significant challenges in the development of new therapies, as drugs must navigate various protective layers at both the tissue and cellular levels to effectively target specific cells.
- A diverse range of therapeutic options is now available, from small molecules to advanced techniques like gene therapies and monoclonal antibodies, with recent innovations rapidly transforming treatment approaches.
- Notable advancements, such as the FDA approval of new RNA-based therapies and viral-vector gene therapies for hemophilia, exemplify the potential for innovative medicines to address complex diseases and improve patient care.
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