المؤلفون: Rocca, Celine J, Rainaldi, Joseph N, Sharma, Jay, Shi, Yanmeng, Haquang, Joseph H, Luebeck, Jens, Mali, Prashant, Cherqui, Stephanie

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Biotechnology, Stem Cell Research, Pediatric, Neurosciences, Transplantation, Regenerative Medicine, Neurodegenerative, Clinical Research, Stem Cell Research - Nonembryonic - Human, Rare Diseases, Genetics, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Aetiology, 2.1 Biological and endogenous factors, Neurological, CRISPR/Cas9, Friedreich's ataxia, P53, ex vivo gene therapy, frataxin, gene editing, hematopoietic stem cells, mitochondrial disorder, neurodegenerative disorder, Medical biotechnology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/8wk3z2ng

المؤلفون: Toshiki Tsunogai, Toya Ohashi, Yohta Shimada, Takashi Higuchi, Ayaka Kimura, Ayako M. Watabe, Fusao Kato, Hiroyuki Ida, Hiroshi Kobayashi

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 448-460 (2022)

مصطلحات موضوعية: GM1-gangliosidosis, ex vivo gene therapy, lentiviral vector, lysosomal storage disorder, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122000602; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/7324db0761c5449a83b52405a909083c

المؤلفون: Masayuki Aso, Tokuo T. Yamamoto, Masayuki Kuroda, Jun Wada, Yoshitaka Kubota, Ko Ishikawa, Yoshiro Maezawa, Naoya Teramoto, Ayako Tawada, Sakiyo Asada, Yasuyuki Aoyagi, Mika Kirinashizawa, Akinobu Onitake, Yuta Matsuura, Kunio Yasunaga, Shun-ichi Konno, Katsuaki Nishino, Misato Yamamoto, Junko Miyoshi, Norihiko Kobayashi, Masami Tanio, Takayuki Ikeuchi, Hidetoshi Igari, Nobuyuki Mitsukawa, Hideki Hanaoka, Koutaro Yokote, Yasushi Saito

المصدر: Heliyon, Vol 8, Iss 11, Pp e11271- (2022)

مصطلحات موضوعية: Cholesterol homeostasis, Ex vivo gene therapy, Familial LCAT deficiency, HDL, LCAT, Proteinuria, Science (General), Q1-390, Social sciences (General), H1-99

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2405844022025592; https://doaj.org/toc/2405-8440

URL الوصول: https://doaj.org/article/d81d0aaee5b947aba70e26e8afc408b1

المؤلفون: Masayuki Kuroda, Makoto Hori, Yoshiro Maezawa, Yoshitaka Kubota, Nobuyuki Mitsukawa, Yuki Shiko, Yoshihito Ozawa, Yohei Kawasaki, Yasushi Saito, Hideki Hanaoka, Koutaro Yokote

المصدر: Contemporary Clinical Trials Communications, Vol 28, Iss , Pp 100946- (2022)

مصطلحات موضوعية: Familial lecithin:cholesterol acyltransferase deficiency, ex vivo gene therapy, Adipocytes, Regenerative medicine, Enzyme replacement therapy, Medicine (General), R5-920

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2451865422000631; https://doaj.org/toc/2451-8654

URL الوصول: https://doaj.org/article/0b3eb7af54c9449696aeeda1d7be6bf9

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المؤلفون: Argyrw Niti, Georgios Koliakos, Anna Michopoulou

المصدر: Bioengineering, Vol 10, Iss 4, p 422 (2023)

مصطلحات موضوعية: epidermolysis bullosa, stem cells, ex vivo gene therapy, Technology, Biology (General), QH301-705.5

وصف الملف: electronic resource

Relation: https://www.mdpi.com/2306-5354/10/4/422; https://doaj.org/toc/2306-5354

URL الوصول: https://doaj.org/article/7c6fb977c4d44ad19782a19fc7190633

المؤلفون: Rustem R Islamov, Farid V Bashirov, Mikhail E Sokolov, Andrei A Izmailov, Filip O Fadeev, Vage A Markosyan, Maria A Davleeva, Olga V Zubkova, Maxim M Smarov, Denis Yu Logunov, Boris S Naroditskyi, Ilnur I Salafutdinov, Albert A Rizvanov, Ramil G Turaev

المصدر: Neural Regeneration Research, Vol 16, Iss 2, Pp 357-361 (2021)

مصطلحات موضوعية: chimeric ad5/35f virus, enhanced green fluorescent protein, gene-modified leucoconcentrate, mini pig, peripheral blood, personalized ex vivo gene therapy, plastic blood bag, spinal cord injury, Neurology. Diseases of the nervous system, RC346-429

وصف الملف: electronic resource

Relation: http://www.nrronline.org/article.asp?issn=1673-5374;year=2021;volume=16;issue=2;spage=357;epage=361;aulast=Islamov; https://doaj.org/toc/1673-5374

URL الوصول: https://doaj.org/article/78d62d7f6cd547bebfb841d7401a14b7

المؤلفون: Miho Wada, Yohta Shimada, Sayoko Iizuka, Natsumi Ishii, Hiromi Hiraki, Toshiaki Tachibana, Kazuhiro Maeda, Mitsuru Saito, Shoutaro Arakawa, Takuya Ishimoto, Takayoshi Nakano, Hiroyuki Ida, Toya Ohashi, Hiroshi Kobayashi

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 261-274 (2020)

مصطلحات موضوعية: mucopolysaccharidosis type II, bone lineage, bone mineral density, bone strength, ex vivo gene therapy, lentivirus, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120301972; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/d2d11fb78c1542e5850cea2f318246be

المؤلفون: Morita, Masashi^Aff3

المساهمون: Imanaka, Tsuneo, editor^Aff1, Shimozawa, Nobuyuki, editor^Aff2

المصدر: Peroxisomes: Biogenesis, Function, and Role in Human Disease. :171-200

المؤلفون: Hoogendoorn, Karin H.^Aff4

المساهمون: Crommelin, Daan J. A., editor^Aff1, Sindelar, Robert D., editor^Aff2, Meibohm, Bernd, editor^Aff3

المصدر: Pharmaceutical Biotechnology : Fundamentals and Applications. :357-402