Results 21 to 30 of about 824,852 (209)

CRISPR medicine for blood disorders: Progress and challenges in delivery

Frontiers in Genome Editing, 2023
Blood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical
Tahereh Mohammadian Gol, Guillermo Ureña-Bailén, Yujuan Hou, Ralph Sinn, Justin S. Antony, Rupert Handgretinger, Rupert Handgretinger, Markus Mezger   +7 more
doaj   +1 more source

Recent progress in microRNA-based delivery systems for the treatment of human disease [PDF]

ExRNA, 2019
MicroRNAs (miRNAs) are naturally occurring, small non-coding RNAs that mediate posttranscriptional regulation. Based on the level of sequence complementarity, miRNAs lead to the degradation of target mRNAs or the suppression of mRNA translation, thereby ...
Yong Fu, Jiangning Chen, Zhen Huang
doaj   +1 more source

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]

, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Amit Nathwani, Baker, AH, Lorraine M Work, Tracey Graham, McIntosh, JB, Jenny McIntosh, Nathwani, A, Work, LM, Graham, T, McIntosh, J, Andrew H Baker   +10 more
core   +1 more source

Differential Transmission of Antiviral Drug-Resistant Chikungunya Viruses by Aedes Mosquitoes

mSphere, 2018
The chikungunya virus (CHIKV) is transmitted by female Aedes aegypti and Aedes albopictus mosquitoes, mostly present in (sub)tropical regions. No antivirals are available to treat CHIKV infections.
Leen Delang, Pei-Shi Yen, Thomas Vallet, Marie Vazeille, Marco Vignuzzi, Anna-Bella Failloux   +5 more
doaj   +1 more source

Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]

, 2004
BACKGROUND: Inactivating genes in vivo is an important technique for establishing their function in the adult nervous system. Unfortunately, conventional knockout mice may suffer from several limitations including embryonic or perinatal lethality and the
Hermens, Wim T J M C, Lieberman, A., Eggers, R., Verhaagen, J., Sablitzky, Fred, Chakravarthy, Sridhara, Zhang, Jing Ying, Zhang, J.Y., Hermens, Wim TJMC, Zhang, Jing, Verhaagen, J, Ahmed, Bushra, Anderson, P.N., Eggers, R, Eggers, Ruben, Levelt, Christiaan, Chakravarthy, S, Levelt, C., Ahmed, BY, Verhaagen, Joost, Lieberman, A.R., Hermens, WTJMC, Lieberman, AR, Niclou, Simone P, Anderson, Patrick N., Ahmed, Bushra Y, Hermens, W.T., Niclou, SP, Anderson, PN, NICLOU, Simone P., Lieberman, A R, Zhang, JY, Anderson, Patrick N, Sablitzky, F., Levelt, C, Sablitzky, F, Niclou, S.P., Lieberman, A Robert, Chakravarthy, S., Ahmed, B.Y.   +39 more
core   +1 more source

Development of Robust Freeze-Drying Process for Long-Term Stability of rVSV-SARS-CoV-2 Vaccine

Viruses
The thermostability of vaccines, particularly enveloped viral vectored vaccines, remains a challenge to their delivery wherever needed. The freeze-drying of viral vectored vaccines is a promising approach but remains challenging due to the water removal ...
MD Faizul Hussain Khan, Maryam Youssef, Sean Nesdoly, Amine A. Kamen   +3 more
doaj   +1 more source

Rapid In-Process Monitoring of Lentiviral Vector Particles by High-Performance Liquid Chromatography

Molecular Therapy: Methods & Clinical Development, 2020
Lentiviral vectors (LVs) are a popular gene delivery tool in cell and gene therapy and they are a primary tool for ex vivo transduction of T cells for expression of chimeric antigen receptor (CAR) in CAR-T cell therapies.
Julia Transfiguracion, Michelle Yen Tran, Stéphane Lanthier, Sonia Tremblay, Nathalie Coulombe, Mauro Acchione, Amine A. Kamen   +6 more
doaj   +1 more source

Viral Vectors Applied for RNAi-Based Antiviral Therapy

, 2020
RNA interference (RNAi) provides the means for alternative antiviral therapy. Delivery of RNAi in the form of short interfering RNA (siRNA), short hairpin RNA (shRNA) and micro-RNA (miRNA) have demonstrated efficacy in gene silencing for therapeutic ...
Kenneth Lundstrom
core   +1 more source

Are Viral Vectors Any Good for RNAi Antiviral Therapy?

, 2020
RNA interference (RNAi) represents a novel approach for alternative antiviral therapy. However, issues related to RNA delivery and stability have presented serious obstacles for obtaining good therapeutic efficacy.
Kenneth Lundstrom
core   +1 more source

Analysis of Adenovirus-Host Interactions to Improve Recombinant Adenoviral Vectors for Gene Therapy [PDF]

, 2011
Recombinant adenoviral vectors are among the most commonly used vehicles in gene therapy. Replication-deficient adenoviruses include early generation adenoviruses, which are deleted in less than three adenoviral genes, and the high-capacity adenoviruses (
Rauschhuber, Christina
core   +1 more source