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    Academic Journals

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• Antiviral immunity and nucleic acid sensing in haematopoietic stem cell gene engineering.

  Authors:Francesco Piras and Anna Kajaste-RudnitskiFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 202110,377 words Report 1630LThe low gene manipulation efficiency of human hematopoietic stem and progenitor cells (HSPC) remains a major hurdle for sustainable and broad clinical application of innovative therapies for a wide range of disorders....

• miR-19a/19b improves the therapeutic potential of mesenchymal stem cells in a mouse model of myocardial infarction.

  Authors:Chengbo Chen, Tianbao Chen, Yangyi Li, and Youbang XuFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20214,447 words Report 1330LMyocardial infarction (MI) is the cardiac emergency that may leads to myocardial necrosis. Mesenchymal stem cells (MSCs) could be used to induce myocardial differentiation. However, the efficiency remains low. The aim of...

• Advances in genome editing: the technology of choice for precise and efficient [beta]-thalassemia treatment.

  Authors:Gibran Ali, Muhammad Akram Tariq, Kamran Shahid, Fridoon Jawad Ahmad, and Javed AkramFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20217,213 words Report 1530LBeta ([beta])-thalassemia is one of the most significant hemoglobinopathy worldwide. The high prevalence of the [beta]-thalassemia carriers aggravates the disease burden for patients and national economies in the...

• Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype.

  Author:Zoltán IvicsFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20212,735 words Article 1600LAuthor(s): Zoltán Ivics 1 Author Affiliations: (1) Transposition and Genome Engineering, Division of Medical Biotechnology, Paul Ehrlich Institute, Langen, Germany Several clinical studies have demonstrated that...

• Particulate mediators of the bystander effect linked to suicide and interferon-[beta] transgene expression in melanoma cells.

  Authors:Lucrecia Agnetti, Chiara Fondello, María Florencia Arbe, Gerardo C. Glikin, and Liliana M. E. FinocchiaroFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 202110,158 words Report 1480LIn the context of comparative oncology, melanoma cells derived from companion animal tumors are good models for optimizing and predicting their in vivo response to therapeutic strategies. Here, we report that human,...

• Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples.

  Authors:Shirin R. Modarai, Sambee Kanda, Kevin Bloh, Lynn M. Opdenaker, and Eric B. KmiecFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20215,602 words Report 1570LClustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and their associated CRISPR-associated nucleases (Cas) are among the most promising technologies for the treatment of hemoglobinopathies including Sickle...

• Racism in science: a perspective from Gene Therapy.

  Author:Janine ScholefieldFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20211,231 words Editorial 1210LAuthor(s): Janine Scholefield 1 2 Author Affiliations: (1) Bioengineering and Integrated Genomics Research Group, NextGen Health Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa (2)...

• The stability of envelope-pseudotyped lentiviral vectors.

  Authors:Iris J. C. Dautzenberg, Martijn J. W. E. Rabelink, and Rob C. HoebenFrom:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 202111,385 words Report 1500LLentiviral vectors have become popular tools for stable genetic modification of mammalian cells. In some applications of lentiviral vector-transduced cells, infectious-lentiviral particles should be absent....

• Maximizing lentiviral vector gene transfer in the CNS.

  Authors:Morgane Humbel, Mergim Ramosaj, Virginie Zimmer, Sara Regio, Ludiwine Aeby, Sylvain Moser, et al.From:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 20219,266 words Report 1410LGene transfer is a widely developed technique for studying and treating genetic diseases. However, the development of therapeutic strategies is challenging, due to the cellular and functional complexity of the central...

• Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters.

  Authors:Bart Nieuwenhuis, Barbara Haenzi, Sam Hilton, Alejandro Carnicer-Lombarte, Barbara Hobo, Joost Verhaagen, et al.From:Gene Therapy (Vol. 28, Issue 1-2) Peer-Reviewed

  Feb. 202112,019 words Report 1530LAdeno-associated viral vectors are widely used as vehicles for gene transfer to the nervous system. The promoter and viral vector serotype are two key factors that determine the expression dynamics of the transgene. A...