Ex Vivo Gene Therapy

Baranyi, Lajos; Slepushkin, Vladimir; Dropulić, Boro

doi:10.1016/b978-0-12-394295-1.00001-9

Cited by 4 publications

(5 citation statements)

References 210 publications

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“…Several groups have used the Sleeping Beauty method to introduce chimeric antigen receptors [59] and gene imaging agents [60]. A more comprehensive description of these vectors are provided in other reviews [4,61,62].…”

Section: Nonviral Vectorsmentioning

confidence: 99%

“…Artificial signaling cascade introduced into T cells allow for Boolean decision making [38] Genome editing Transgene sequences can be inserted to modify the genomes of cells (e.g. CRISPR), knocking down key components [4] Knockdown of CCR5 to confer resistance to HIV in HSC [39] deaminase into HSC to render the descendant T cells functional has led to successful treatment of X-linked severe combined immunodeficiency [41]. Another reason for introducing transgenes is to track infused cells.…”

Section: Chemotaxis Of Mammalian Cells Towards An Inert Signal Done Bmentioning

confidence: 99%

“…Lentiviral vectors offer an attractive alternative to retroviral vectors, and their use in gene-modified cell therapies and gene therapies is rapidly increasing [50], arguably now the preferred delivery platform for gene modified cells [4]. They confer a lower risks of insertional mutagenesis; their complex structures allow for bigger payloads, and they infect both dividing and nondividing cells (retroviruses only infect actively dividing cells).…”

Section: Lentiviral Vectorsmentioning

confidence: 99%

“…Much of this development demands the creation of gene-modified cells, now made more accessible to the clinic through improved editing and vector delivery platforms. Cell-based gene therapy represents a unique sophisticated delivery system: off-target effects can be avoided by ex vivo gene modification and adequate monitoring and characterization of transgene expression are made possible in the controlled confines of the laboratory [4]. Inserted genes can be used to control cell behavior providing hitherto unprecedented versatility and safety.…”

Section: Cell Therapy-a New Therapeutic Pillar Of Medicine?mentioning

confidence: 99%

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Gene-modified, cell-based therapies—an overview

2016

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Section: Nonviral Vectorsmentioning

confidence: 99%

Section: Chemotaxis Of Mammalian Cells Towards An Inert Signal Done Bmentioning

confidence: 99%

Section: Lentiviral Vectorsmentioning

confidence: 99%

Section: Cell Therapy-a New Therapeutic Pillar Of Medicine?mentioning

confidence: 99%

See 2 more Smart Citations

Gene-modified, cell-based therapies—an overview

2016

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“…Alternative, RNAi technology has demonstrated substantial efficacy, in the accelerated analysis of gene function across extensive gene pools within a diverse array of organisms [7]. Despite its advantages, RNAi technology is contingent upon the RNA-induced silencing complex (RISC), presenting constraints such as a truncated half-life and the intricate delivery of RNAi molecules [8]. Additionally, these methodologies lack the capacity for remote spatio-temporal modulation of gene interference within living plant cellular systems.…”

Section: Introductionmentioning

confidence: 99%

Design of a nanobiosystem with remote photothermal gene silencing in Chlamydomonas reinhardtii to increase lipid accumulation and production

et al. 2023

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Research development in the precise control of gene expression in plant cells is an emerging necessity that would lead to the elucidation of gene function in these biological systems. Conventional gene-interfering techniques, such as micro-RNA and short interfering RNA, have limitations in their ability to downregulate gene expression in plants within short time periods. However, nanotechnology provides a promising new avenue with new tools to overcome these challenges. Here, we show that functionalized gold nanoparticles, decorated with sense and antisense oligonucleotides (FANSAO), can serve as a remote-control optical switch for gene interference in photosynthetic plant cells. We demonstrate the potential of employing LEDs as optimal light sources to photothermally dehybridize the oligonucleotides on the surface of metallic nanostructures, consequently inducing regulation of gene expression in plant cells. We show the efficiency of metallic nanoparticles in absorbing light from an LED source and converting it to thermal energy, resulting in a local temperature increase on the surface of the gold nanoparticles. The antisense oligonucleotides are then released due to the opto-thermal heating of the nanobiosystem composed of the metallic nanoparticles and the sense-antisense oligonucleotides. By applying this approach, we silenced the Carnitine Acyl Carnitine Translocase genes at 90.7%, resulting in the accumulation of lipid bodies in microalgae cells. These results exhibit the feasibility of using functionalized gold nanoparticles with sense and antisense oligonucleotides to enhance nucleic acid delivery efficiency and, most importantly, allow for temporal control of gene silencing in plant cells. These nanobiosystems have broad applications in the development and biosynthesis of biofuels, pharmaceuticals, and specialized chemicals.

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National guidelines for gene therapy product (2019)

Sivagourounadin

Ravichandran

Rajendran

2021

Perspectives in Clinical Research

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The “National Guidelines for Gene Therapy Product (GTP) Development and Clinical Trials” prepared by the Indian Council of Medical Research and Department of Biotechnology in 2019 came as a welcome step in the process of regulation of gene therapy research, as there was a lack of Indian guidelines earlier specific to gene therapy. Indian researchers have taken their step in setting the path of gene therapy research, and this guideline serves to provide the standards starting from its development up to translation to new drug including the ethical, scientific, and regulatory requirements to be followed during the conduct of trial. The Indian guidelines were framed with reference to United States-Food and Drug Administration and European Union guidelines on gene therapy. It is the responsibility of all the stakeholders involved in the development of GTP to adhere to the national guidelines. This review provides an outline of the Indian regulatory guidelines on GTP.

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Ex Vivo Gene Therapy

Cited by 4 publications

References 210 publications

Gene-modified, cell-based therapies—an overview

Gene-modified, cell-based therapies—an overview

Design of a nanobiosystem with remote photothermal gene silencing in Chlamydomonas reinhardtii to increase lipid accumulation and production

National guidelines for gene therapy product (2019)

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