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Showing 20 of 29 academic articles

Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial

Andrew D. Leavitt, Johnny Mahlangu, Priyanka Raheja, Emily Symington et al. • Research and Practice in Thrombosis and Haemostasis (2024)

Background: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection. Objectives: Determine valoctocogene roxaparvovec du...

DOAJ Open Access (DOAJ) DOI: 10.1016/j.rpth.2024.102615

Broad-Spectrum Antiviral Activity of an Ankyrin Repeat Protein on Viral Assembly against Chimeric NL4-3 Viruses Carrying Gag/PR Derived from Circulating Strains among Northern Thai Patients

Supachai Sakkhachornphop, Sudarat Hadpech, Tanchanok Wisitponchai, Chansunee Panto et al. • Viruses (2018)

Certain proteins have demonstrated proficient human immunodeficiency virus (HIV-1) life cycle disturbance. Recently, the ankyrin repeat protein targeting the HIV-1 capsid, AnkGAG1D4, showed a negative effect on the viral assembly of the HIV...

DOAJ Open Access (DOAJ) DOI: 10.3390/v10110625

Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine

Ruixuan Zheng, Lexiang Zhang, Rokshana Parvin, Lihuang Su et al. • Advanced Science (2023)

Abstract Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions o...

DOAJ Open Access (DOAJ) DOI: 10.1002/advs.202300195

Characterization data on the topical carrier DDC642

Eline Desmet, Stefanie Bracke, Katrien Forier, Lien Taevernier et al. • Data in Brief (2016)

This article contains original data, figures and methods used in the characterization of the liposomal carrier ‘DDC642’ for topical applications, described in “An elastic liposomal formulation for RNAi-based topical treatment of skin disord...

DOAJ Open Access (DOAJ) DOI: 10.1016/j.dib.2016.03.091

Application of Gene Therapy to Oral Diseases

Seiichi Yamano, Kenji Inoue, Yoichiro Taguchi • Pharmaceutics (2025)

Gene therapy has emerged as a promising therapeutic approach across various oral diseases. This review examines current applications and future prospects of gene therapy in dentistry, focusing on five key areas: oral cancer, cancer-related ...

DOAJ Open Access (DOAJ) DOI: 10.3390/pharmaceutics17070859

Activation of neuronal genes via LINE-1 elements upon global DNA demethylation in human neural progenitors

Marie E Jönsson, Per Ludvik Brattås, Charlotte Gustafsson, Rebecca Petri et al. • Nature Communications (2019)

DNA methylation plays an important role in silencing transposable elements. Here the authors find that loss of DNMT1 and DNA methylation leads to transcriptional activation and chromatin remodelling of evolutionarily young—hominoid-specific...

DOAJ Open Access (DOAJ) DOI: 10.1038/s41467-019-11150-8

Evaluation of the SH-SY5Y cell line as an in vitro model for potency testing of a neuropeptide-expressing AAV vector

Jeanette Zanker, Daniela Hüser, Adrien Savy, Sara Lázaro-Petri et al. • Frontiers in Molecular Neuroscience (2023)

Viral vectors have become important tools for basic research and clinical gene therapy over the past years. However, in vitro testing of vector-derived transgene function can be challenging when specific post-translational modifications are...

DOAJ Open Access (DOAJ) DOI: 10.3389/fnmol.2023.1280556

Genomic sciences and medical biotechnology

Govindarajan Padmanaban • NOT FOUND REPOSITORY (Indian Institute of Science Bangalore) (2008)

The advent of genomic sciences has provided enormous information on the gene content and its expression in a wide variety of organisms, including the human. This has paved the way for understanding the differences between normal and abnorma...

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International Journal of Medical Biotechnology & Genetics

Saket RD, Kol S, Tripathi AK, Singh SP et al. • International Journal of Medical Biotechnology & Genetics (2022)

International Journal of Medical Biotechnology & Genetics (IJMBG) ISSN: 2379-1020 is a comprehensive, peer reviewed journal devoted to Medical Biotechnology and Genetics. publishes original research, latest developments, review papers, scie...

OPENALEX cc-by DOI: https://doi.org/10.19070/2379-1020