A Gene Therapy Breakthrough Could Offer a Treatment for the Rare and Deadly ‘Bubble Boy’ Disease

Researchers used an experimental gene therapy to develop a possible treatment for a rare and deadly immune disorder known as “bubble boy” disease, the National Institutes of Health (NIH) announced Wednesday. Because of a gene mutation, babies who are born with X-linked severe combined immunodeficiency (X-SCID) do not develop immune cells properly, leaving them highly susceptible to infections. The condition, which strikes up to one in 50,000 newborns, primarily affects boys and requires extreme measures to prevent infection. In one famous case, a boy with SCID, David Vetter, lived in a sterile plastic “bubble” until his death at age 12 — hence the disease’s nickname. Until now, the best treatment for these difficult cases relied on using bone marrow transplants from tissue-matched siblings to restore immunity. But since many patients do not have a sibling who’s a match, doctors often resort to other donors. Such donations can improve patients’ health, but they often can’t produce full immunity, the NIH says — which is why gene therapy has seemed so promising. Still, previous gene therapy techniques either did not fully restore immune function or resulted in side effects as serious as leukemia. Now, a gene therapy advance described in a small New England Journal of Medicine study seems to have safely produced a robust immune response in eight young patients, offering families and doctors new hope. Eight infants with...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Research Source Type: news

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Publication date: Available online 4 April 2020Source: Journal of Geriatric OncologyAuthor(s): Kelly M. Trevino, Amy Stern, Holly G. Prigerson
Source: Journal of Geriatric Oncology - Category: Cancer & Oncology Source Type: research
Publication date: Available online 4 April 2020Source: Journal of Geriatric OncologyAuthor(s): Claire A. Surr, Rachael Kelley, Alys W. Griffiths, Laura Ashley, Fiona Cowdell, Ann Henry, Michelle Collinson, Ellen Mason, Amanda J. Farrin
Source: Journal of Geriatric Oncology - Category: Cancer & Oncology Source Type: research
Publication date: Available online 12 March 2020Source: The Journal of Allergy and Clinical Immunology: In PracticeAuthor(s): Karsten Weller, Tamara Donoso, Markus Magerl, Emel Aygören-Pürsün, Petra Staubach, Inmaculada Martinez-Saguer, Tomasz Hawro, Sabine Altrichter, Karoline Krause, Frank Siebenhaar, Martin Metz, Torsten Zuberbier, Denise Freier, Marcus Maurer
Source: The Journal of Allergy and Clinical Immunology: In Practice - Category: Allergy & Immunology Source Type: research
The agency granted the authorization to global biopharacuetical company Cellex. Of 128 samples confirmed positive by RT-PCR in premarket testing, 120 tested positive by IgG, IgM, or both.FDA Approvals
Source: Medscape Allergy Headlines - Category: Allergy & Immunology Tags: Infectious Diseases News Alert Source Type: news
Publication date: July 2020Source: Materials Science and Engineering: C, Volume 112Author(s): Yuquan Li, Tongmeng Jiang, Li Zheng, Jinmin Zhao
Source: Materials Science and Engineering: C - Category: Materials Science Source Type: research
Abstract OBJECTIVES: To identify and discuss scientific evidence of the effects of ginger use on the management of chemotherapy-induced nausea and vomiting. METHODS: This is an integrative reviewperformed by Ganong's reference. RESULTS: We included 24 studies, highlighting three thematic categories, namely 1) antiemetic action of ginger - nausea (13 articles; of these, nine significant) and emesis (10 studies; of these, six significant); 2) action in the control of nausea (11 articles; of these, six significant) and vomiting (8 articles; of these, three significant) in the acute phase; 3) action in the c...
Source: Revista Brasileira de Enfermagem - Category: Nursing Authors: Tags: Rev Bras Enferm Source Type: research
Connection between SOX7 Expression and Breast Cancer Prognosis. Med Sci Monit. 2020 Apr 02;26:e921510 Authors: Qin CX, Yang XQ, Zhan ZY Abstract BACKGROUND SOX7 exerts a repressing effect against tumors and imposes vital influences on malignancies. Our research discussed the importance of SOX7 in breast cancer prognoses. MATERIAL AND METHODS SOX7 mRNA expression in breast cancer tissues samples and matched adjacent normal controls of breast cancer patients was measured by quantitative real-time-polymerase chain reaction (qRT-PCR). The relationship of SOX7 with clinicopathological characteristics w...
Source: Medical Science Monitor - Category: Research Tags: Med Sci Monit Source Type: research
CONCLUSIONS These 7 DEGs might be potential diagnostic and prognostic biomarkers for PAC and help uncovering the mechanism of PAC. PMID: 32235821 [PubMed - in process]
Source: Medical Science Monitor - Category: Research Tags: Med Sci Monit Source Type: research
CONCLUSION: Fewer eligible donors in spite of an increase in brain-dead potential organ donors suggests that reduction in these donations resulted mainly from factors associated with family consent. Among such factors, implementation of the Life-sustaining Treatment Act appears to be most important. Abolition of family compensation and the incident in which a brain-dead donor's remains were mistreated may also have influenced family consent. PMID: 32242345 [PubMed - in process]
Source: J Korean Med Sci - Category: General Medicine Authors: Tags: J Korean Med Sci Source Type: research
Publication date: Available online 3 April 2020Source: Food BioscienceAuthor(s): M.S. Taniya, Reshma MV, Shanimol PS, Gayatri Krishnan, Priya S
Source: Food Bioscience - Category: Food Science Source Type: research
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