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Advanced Search Results For "GENE therapy"

1 - 10 of 195,063 results for
 "GENE therapy"
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The steep uphill path leading to ex vivo gene therapy for genodermatoses.

Publication Type: Academic Journal

Source(s): American journal of physiology. Cell physiology [Am J Physiol Cell Physiol] 2022 Sep 01; Vol. 323 (3), pp. C896-C906. Date of Electronic Publication: 2022 Aug 01.

Abstract: Cell therapy, gene therapy, and tissue engineering have the potential to revolutionize the field of regenerative medicine. In particular, gene therapy is understood as the therapeutical correction of mutated genes by addition of a correct copy of the g...

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Schwannoma Gene Therapy via Adeno-Associated Viral Vector Delivery of Apoptosis-Associated Speck-like Protein Containing CARD (ASC): Preclinical Efficacy and Safety.

Publication Type: Academic Journal

Source(s): International journal of molecular sciences [Int J Mol Sci] 2022 Jan 13; Vol. 23 (2). Date of Electronic Publication: 2022 Jan 13.

Abstract: Schwannomas are tumors derived from Schwann-lineage cells, cells that protect and support myelinated nerves in the peripheral nervous system. They are typically slow-growing, encapsulated and benign. These tumors develop along peripheral, spinal and cr...

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Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.

Publication Type: Academic Journal

Source(s): Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2021 Nov 03; Vol. 29 (11), pp. 3205-3218. Date of Electronic Publication: 2021 Sep 10.

Abstract: Hematopoietic stem and progenitor cell (HSPC) gene therapies have recently moved beyond gene-addition approaches to encompass targeted genome modification or correction, based on the development of zinc finger nucleases (ZFNs), transcription activator-...

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Genome editing in large animal models.

Publication Type: Academic Journal

Source(s): Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2021 Nov 03; Vol. 29 (11), pp. 3140-3152. Date of Electronic Publication: 2021 Oct 01.

Abstract: Although genome editing technologies have the potential to revolutionize the way we treat human diseases, barriers to successful clinical implementation remain. Increasingly, preclinical large animal models are being used to overcome these barriers. In...

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Bacteriophage-Mediated Cancer Gene Therapy.

Publication Type: Academic Journal

Source(s): International journal of molecular sciences [Int J Mol Sci] 2022 Nov 17; Vol. 23 (22). Date of Electronic Publication: 2022 Nov 17.

Abstract: Bacteriophages have long been considered only as infectious agents that affect bacterial hosts. However, recent studies provide compelling evidence that these viruses are able to successfully interact with eukaryotic cells at the levels of the binding,...

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Effective therapies for sickle cell disease: are we there yet?

Publication Type: Academic Journal

Source(s): Trends in genetics : TIG [Trends Genet] 2022 Dec; Vol. 38 (12), pp. 1284-1298. Date of Electronic Publication: 2022 Aug 04.

Abstract: Sickle cell disease (SCD) is a common genetic blood disorder associated with acute and chronic pain, progressive multiorgan damage, and early mortality. Recent advances in technologies to manipulate the human genome, a century of research and the devel...

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Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?

Publication Type: Academic Journal

Source(s): Human gene therapy [Hum Gene Ther] 2022 Nov; Vol. 33 (21-22), pp. 1109-1120. Date of Electronic Publication: 2022 Nov 04.

Abstract: Advances in gene therapy, synthetic biology, cancer genomics, and patient-derived cancer models have expanded the repertoire of strategies for targeting human cancers using viral vectors. Novel capsids, synthetic promoters, and therapeutic payloads are...

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Application of Gene Therapy in Hemophilia.

Publication Type: Academic Journal

Source(s): Current medical science [Curr Med Sci] 2022 Oct; Vol. 42 (5), pp. 925-931. Date of Electronic Publication: 2022 Oct 19.

Authors:

Abstract: Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy. It holds out hope of a cure for single-gene genetic diseases such a...

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Runx1 Messenger RNA Delivered by Polyplex Nanomicelles Alleviate Spinal Disc Hydration Loss in a Rat Disc Degeneration Model.

Publication Type: Academic Journal

Source(s): International journal of molecular sciences [Int J Mol Sci] 2022 Jan 05; Vol. 23 (1). Date of Electronic Publication: 2022 Jan 05.

Abstract: Vertebral disc degenerative disease (DDD) affects millions of people worldwide and is a critical factor leading to low back and neck pain and consequent disability. Currently, no strategy has addressed curing DDD from fundamental aspects, because the p...

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Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity.

Publication Type: Academic Journal

Source(s): Hematology/oncology clinics of North America [Hematol Oncol Clin North Am] 2022 Aug; Vol. 36 (4), pp. 829-851. Date of Electronic Publication: 2022 Jun 29.

Abstract: The field of gene therapy has experienced tremendous growth in the last decade ranging from improvements in the design of viral vectors for gene addition of therapeutic gene cassettes to the discovery of site-specific nucleases targeting transgenes to ...

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