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Gene therapy for inherited immunodeficiency

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Julie Brault, Guillaume Vaganay, Aline Le Roy, Jean-Luc Lenormand, Sandra Cortes & Marie José Stasia. (2017) Therapeutic effects of proteoliposomes on X-linked chronic granulomatous disease: proof of concept using macrophages differentiated from patient-specific induced pluripotent stem cells. International Journal of Nanomedicine 12, pages 2161-2177.
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Iman H Nasr, Ania L Manson, Humaid A Al Wahshi & Hilary J Longhurst. (2016) Optimizing hereditary angioedema management through tailored treatment approaches. Expert Review of Clinical Immunology 12:1, pages 19-31.
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Sem J. Aronson, Ulrich Beuers & Piter J. Bosma. (2015) Progress and challenges in gene therapy for Crigler–Najjar syndrome. Expert Opinion on Orphan Drugs 3:12, pages 1387-1396.
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Antoine N Saliba, Raafat S Alameddine, Afif R Harb & Ali T Taher. (2015) Globin gene regulation for treating β-thalassemias: progress, obstacles and future. Expert Opinion on Orphan Drugs 3:9, pages 1047-1062.
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Bruce Levine, Rachel Leskowitz & Megan Davis. (2015) Personalized gene therapy locks out HIV, paving the way to control virus without antiretroviral drugs. Expert Opinion on Biological Therapy 15:6, pages 831-843.
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Martha S Field, Elena Kamynina, David Watkins, David S Rosenblatt & Patrick J Stover. (2015) New insights into the metabolic and nutritional determinants of severe combined immunodeficiency. Rare Diseases 3:1.
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Toru Uchiyama, Toshinao Kawai, Kazuhiko Nakabayashi, Yumiko Nakazawa, Fumihiro Goto, Kohji Okamura, Toyoki Nishimura, Koji Kato, Nobuyuki Watanabe, Akane Miura, Toru Yasuda, Yukiko Ando, Tomoko Minegishi, Kaori Edasawa, Marika Shimura, Yumi Akiba, Aiko Sato-Otsubo, Tomoyuki Mizukami, Motohiro Kato, Koichi Akashi, Hiroyuki Nunoi & Masafumi Onodera. (2023) Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD. Molecular Therapy 31:12, pages 3424-3440.
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Sameer Bahal, Klesti Karaxhuku & Giorgia Santilli. (2022) Gene Editing in Human Haematopoietic Stem Cells for the Treatment of Primary Immunodeficiencies. Molecular Diagnosis & Therapy 27:1, pages 15-28.
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Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez, Gayatri Rao, Jonathan D. Schwartz, Ken M. Law, Julián Sevilla, Paula Río, Rosa Yáñez, Juan A. Bueren & Elena Almarza. (2022) Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I. Molecular Therapy - Methods & Clinical Development 26, pages 459-470.
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Toru Uchiyama, Sirirat Takahashi, Kazuhiko Nakabayashi, Kohji Okamura, Kaori Edasawa, Masafumi Yamada, Nobuyuki Watanabe, Emi Mochizuki, Toru Yasuda, Akane Miura, Motohiro Kato, Daisuke Tomizawa, Makoto Otsu, Tadashi Ariga & Masafumi Onodera. (2021) Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones. Molecular Therapy - Methods & Clinical Development 23, pages 424-433.
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Aleksandra Lewandowicz-Uszyńska, Gerard Pasternak, Jerzy Świerkot & Katarzyna Bogunia-Kubik. 2021. Medical and Biomedical Updates. Medical and Biomedical Updates 37 54 .
Amy E. O’Connell. (2019) Primary Immunodeficiency in the NICU. NeoReviews 20:2, pages e67-e78.
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Xiuyan Wang & Isabelle Rivière. 2019. Cell and Gene Therapies. Cell and Gene Therapies 265 288 .
Maria Kanariou, Sofia Tantou, Marianna Tzanoudaki & Lilia Lykopoulou. 2019. Pediatric Immunology. Pediatric Immunology 799 803 .
Joseph P. Cornish & Reed F. Johnson. 2019. Defense Against Biological Attacks. Defense Against Biological Attacks 195 223 .
Martha S. FieldElena KamyninaJames Chon & Patrick J. Stover. (2018) Nuclear Folate Metabolism. Annual Review of Nutrition 38:1, pages 219-243.
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Giulio Cossu, Martin Birchall, Tracey Brown, Paolo De Coppi, Emily Culme-Seymour, Sahra Gibbon, Julian Hitchcock, Chris Mason, Jonathan Montgomery, Steve Morris, Francesco Muntoni, David Napier, Nazanin Owji, Aarathi Prasad, Jeff Round, Prince Saprai, Jack Stilgoe, Adrian Thrasher & James Wilson. (2018) Lancet Commission: Stem cells and regenerative medicine. The Lancet 391:10123, pages 883-910.
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Koichiro Saka, Chen-Yi Lai, Masanori Nojima, Masahiro Kawahara, Makoto Otsu, Hiromitsu Nakauchi & Teruyuki Nagamune. (2017) Dissection of Signaling Events Downstream of the c-Mpl Receptor in Murine Hematopoietic Stem Cells Via Motif-Engineered Chimeric Receptors. Stem Cell Reviews and Reports 14:1, pages 101-109.
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Tayebeh SoheiliAmandine Durand, Fernando E. SepulvedaJulie RivièreChantal Lagresle-Peyrou, Hanem Sadek, Geneviève de Saint BasileSamia Martin, Fulvio Mavilio, Marina Cavazzana & Isabelle André-Schmutz. (2017) Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency. Blood Advances 1:27, pages 2781-2789.
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Nathan Paul Sweeney, Jinhong Meng, Hayley Patterson, Jennifer E. Morgan & Myra McClure. (2017) Delivery of large transgene cassettes by foamy virus vector. Scientific Reports 7:1.
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Nixon Niyonzima, Abigail R. Lambert, Rachel Werther, Harshana De Silva Feelixge, Pavitra Roychoudhury, Alexander L. Greninger, Daniel Stone, Barry L. Stoddard & Keith R. Jerome. (2017) Tuning DNA binding affinity and cleavage specificity of an engineered gene-targeting nuclease via surface display, flow cytometry and cellular analyses. Protein Engineering, Design and Selection 30:7, pages 503-522.
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Xiuyan Wang & Isabelle Rivière. (2017) Genetic Engineering and Manufacturing of Hematopoietic Stem Cells. Molecular Therapy - Methods & Clinical Development 5, pages 96-105.
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Dong Kwang Seo, Jeong Hoon Kim, Joongkee Min, Hyung Ho Yoon, Eun-Sil Shin, Seong Who Kim & Sang Ryong Jeon. (2017) Enhanced axonal regeneration by transplanted Wnt3a-secreting human mesenchymal stem cells in a rat model of spinal cord injury. Acta Neurochirurgica 159:5, pages 947-957.
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Koenraad R. Veys, Mohamed A. Elmonem, Fanny O. Arcolino, Lambertus van den Heuvel & Elena Levtchenko. (2017) Nephropathic cystinosis: an update. Current Opinion in Pediatrics 29:2, pages 168-178.
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H. E. Maunder, J. Wright, B. R. Kolli, C. R. Vieira, T. T. Mkandawire, S. Tatoris, V. Kennedy, S. Iqball, G. Devarajan, S. Ellis, Y. Lad, N. G. Clarkson, K. A. Mitrophanous & D. C. Farley. (2017) Enhancing titres of therapeutic viral vectors using the transgene repression in vector production (TRiP) system. Nature Communications 8:1.
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Susana L. Silva & Ana E. Sousa. (2016) Establishment and Maintenance of the Human Naïve CD4+ T-Cell Compartment. Frontiers in Pediatrics 4.
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Kenzaburo Tani. (2016) Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world. International Journal of Hematology 104:1, pages 42-72.
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John M. Routes. 2016. Murray and Nadel's Textbook of Respiratory Medicine. Murray and Nadel's Textbook of Respiratory Medicine 1624 1638.e4 .
H.H. Arts & Nine V.A.M. Knoers. 2016. Kidney Development, Disease, Repair and Regeneration. Kidney Development, Disease, Repair and Regeneration 275 291 .
Panicos Shangaris & Anna L. David. 2016. Fetal Stem Cells in Regenerative Medicine. Fetal Stem Cells in Regenerative Medicine 361 402 .
Jennifer M. Puck. 2015. Genetic Disorders and the Fetus. Genetic Disorders and the Fetus 755 772 .
Lisa M. Ott de Bruin, Stefano Volpi & Kiran Musunuru. (2015) Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies. Frontiers in Immunology 6.
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Makoto Otsu, Masafumi Yamada, Satoru Nakajima, Miyuki Kida, Yoshihiro Maeyama, Norikazu Hatano, Nariaki Toita, Shunichiro Takezaki, Yuka Okura, Ryoji Kobayashi, Yoshinori Matsumoto, Osamu Tatsuzawa, Fumiko Tsuchida, Shunichi Kato, Masanari Kitagawa, Junichi Mineno, Michael S. Hershfield, Pawan Bali, Fabio Candotti, Masafumi Onodera, Nobuaki Kawamura, Yukio Sakiyama & Tadashi Ariga. (2015) Outcomes in Two Japanese Adenosine Deaminase-Deficiency Patients Treated by Stem Cell Gene Therapy with No Cytoreductive Conditioning. Journal of Clinical Immunology 35:4, pages 384-398.
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M. Otsu. (2015) Perspectives on stem cell gene therapy for genetic disorders. ISBT Science Series 10:S1, pages 231-234.
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Antonia Kwan & Jennifer M. Puck. (2015) History and current status of newborn screening for severe combined immunodeficiency. Seminars in Perinatology 39:3, pages 194-205.
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Timothy M. Cox. (2015) Innovative Treatments for Lysosomal Diseases. Best Practice & Research Clinical Endocrinology & Metabolism 29:2, pages 275-311.
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Antonia Kwan & Jennifer M. Puck. (2014) Newborn Screening for Severe Combined Immunodeficiency. Current Pediatrics Reports 3:1, pages 34-42.
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Hildegard Büning & Ulrich T. Hacker. 2015. DGIM Innere Medizin. DGIM Innere Medizin 1 10 .
Helen Chapel, Johan Prevot, Hubert Bobby Gaspar, Teresa Español, Francisco A. Bonilla, Leire Solis & Josina Drabwell. (2014) Primary Immune Deficiencies - Principles of Care. Frontiers in Immunology 5.
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Aisha V. Sauer, Biagio Di Lorenzo, Nicola Carriglio & Alessandro Aiuti. (2014) Progress in gene therapy for primary immunodeficiencies using lentiviral vectors. Current Opinion in Allergy & Clinical Immunology 14:6, pages 527-534.
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REBECCA N. SPENCER, DAVID J. CARR & ANNA L. DAVID. (2015) GENE THERAPY FOR OBSTETRIC CONDITIONS. Fetal and Maternal Medicine Review 25:3-4, pages 147-177.
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Peter J. Späth. (2014) A glance on recent progresses in diagnosis and treatment of primary immunodeficiencies/ Progrese recente în diagnosticul şi tratamentul imunodeficienţelor primare. Romanian Review of Laboratory Medicine 22:3.
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Cuilian Tao, Yufang Zhu, Xianglan Li & Nobutaka Hanagata. (2014) Magnetic mesoporous silica nanoparticles for CpG delivery to enhance cytokine induction via toll-like receptor 9. RSC Adv. 4:86, pages 45823-45830.
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