Filtered By:
Procedure: Transplants
Therapy: Stem Cell Therapy

This page shows you your search results in order of date.

Order by Relevance | Date

Total 595 results found since Jan 2013.

Impact of newborn screening for SCID on the management of congenital athymia
Newborn screening (NBS) programmes for severe combined immunodeficiency (SCID) facilitate early SCID diagnosis and promote early treatment with haematopoietic stem cell transplantation, resulting in improved clinical outcomes. Infants with congenital athymia are also identified through NBS due to severe T-cell lymphopaenia. With the expanding introduction of NBS programmes, referrals of athymic patients for treatment with thymus transplantation have recently increased at Great Ormond Street Hospital (GOSH), London, United Kingdom.
Source: Journal of Allergy and Clinical Immunology - September 5, 2023 Category: Allergy & Immunology Authors: Evey Howley, Zainab Golwala, Matthew Buckland, Federica Barzaghi, Sujal Ghosh, Scott Hackett, Rosie Hague, Fabian Hauck, Ursula Holzer, Adam Klocperk, Minna Koskenvuo, Nufar Marcus, Antonio Marzollo, Malgorzata Pac, Jan Sinclair, Carsten Speckmann, Maarja Source Type: research

News at a glance: Muscular dystrophy therapy, lab-grown chicken, and humans ’ toll on wildlife
BIOMEDICINE Muscular dystrophy therapy approved The U.S. Food and Drug Administration has approved the first gene therapy for Duchenne muscular dystrophy (DMD), a genetic disease that cripples boys and usually results in death by age 30. The treatment from Sarepta Therapeutics introduces a short version of the gene for dystrophin, a crucial muscle protein, which is mutated in patients with DMD. A one-time intravenous infusion of a virus delivers the functioning “microdystrophin” gene into patients’ muscle cells. The 22 June approval is only for boys 4 to 5 years old, a group that appeared likely to ben...
Source: Science of Aging Knowledge Environment - June 29, 2023 Category: Geriatrics Source Type: research

Emapalumab as bridge to hematopoietic cell transplant for STAT1 gain-of-function mutations
Gain-of-function (GOF) mutations of transcription factor STAT1 result in overproduction of IFN- α/β, IFN-γ, and IL-27.1 This leads to immunodeficiency, autoimmune disorders, and cerebral aneurysms.2 Hematopoietic cell transplantation (HCT) is curative but has high rates of graft failure (GF), likely owing to increased levels of IFN-γ, which has been implicated in directly inhibiting hemato poietic stem cell function and also induces Fas expression and increased apopotosis.3,4,5 Emapalumab is a human IFN-γ–blocking mAb, approved for treatment of primary hemophagocytic lymphohistiocytosis.
Source: Journal of Allergy and Clinical Immunology - June 26, 2023 Category: Allergy & Immunology Authors: Binni Kunvarjee, Alan Bidgoli, Rebecca Pellett Madan, Esther Vidal, Devin McAvoy, Kinga K. Hosszu, Andromachi Scaradavou, Barbara G. Spitzer, Kevin J. Curran, Maria Cancio, Andrew C. Harris, Richard J. O ’Reilly, Andrew L. Kung, Susan Prockop, Jaap Jan Tags: Correspondence Source Type: research

Invasive Fungal Sinusitis Risk Factors Among Immunosuppressed Hematopoietic Stem Cell Transplant Recipients
Source: International Forum of Allergy and Rhinology - June 1, 2023 Category: Allergy & Immunology Authors: Marie ‐Ange Munyemana, Anupam Pande, Dorina Kallogjeri, Nyssa F. Farrell, John S. Schneider, Peggy Kendall, Lauren T. Roland Tags: RESEARCH NOTE Source Type: research

Reversal of SLE and hemophagocytic lymphohistiocytosis caused by lysinuric protein intolerance through allogeneic hematopoietic stem cell transplantation
Lysinuric protein intolerance (LPI; Online Mendelian Inheritance in Man [OMIM] identifier 222700) is a rare autosomal recessive metabolic disorder caused by mutations in the SLC7A7 gene, which encodes for y+L cationic amino acid (including arginine, ornithine, and lysine) transporter-1 (y+LAT-1) protein.1 The malfunction of y+LAT-1 in polarized cells impairs the intestinal absorption and renal tubular reabsorption of cationic amino acid, resulting in malnutrition and failure to thrive.2 LPI is also classified as an inborn error of immunity owing to the immune dysregulation caused by malfunction of the y+LAT-1 protein, the ...
Source: Journal of Allergy and Clinical Immunology - March 29, 2023 Category: Allergy & Immunology Authors: Yu Zhou, Lan He, Zhongxun Yu, Meiying Quan, Hongmei Song, Chunfu Li Tags: Correspondence Source Type: research

Two cases of transplant-acquired food allergy who developed resensitization after a negative oral food challenge
Cases of food allergy after hematopoietic stem cell and solid organ transplantation in previously nonallergic transplant recipients were reported as transplant-acquired food allergy (TAFA), but information abo...
Source: Allergy, Asthma and Clinical Immunology - March 22, 2023 Category: Allergy & Immunology Authors: Akiko Nakaoka, Takayasu Nomura, Kazuyoshi Ozeki, Tomotaka Suzuki, Shigeru Kusumoto, Shinsuke Iida and Shinji Saitoh Tags: Case report Source Type: research

Autoimmune manifestations in VEXAS: Opportunities for integration and pitfalls to interpretation
VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) is a novel entity manifesting with a multiplicity of clinical features. Somatic mutations of the UBA1 gene in hematopoietic stem cells constitute the genetic basis of VEXAS. As an X-linked disorder, most cases occur in men, classically developing symptoms during the fifth to sixth decade of life. Considering its multidisciplinary nature involving numerous branches of internal medicine, VEXAS has elicited a wide medical interest and several medical conditions have been associated with this disease.
Source: Journal of Allergy and Clinical Immunology - March 20, 2023 Category: Allergy & Immunology Authors: Alessandro Bruno, Carmelo Gurnari, Tobias Alexander, John A. Snowden, Raffaella Greco, Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation Source Type: research

Gene therapy for SCID, now up to 3!
Since 2000, gene therapy has become an option to treat severe combined immunodeficiency (SCID), a rare but lethal condition characterized by fully defective T lymphocyte differentiation caused by pathogenic variants in least 18 genes. The current treatment by allogeneic hematopoietic stem cell transplantation (HSCT) is effective but carries the risk of graft-versus-host disease. This treatment is based on ex  vivo gene transfer by using retroviral vector (initially gammaretrovirus, now lentivirus) to transduce the patient bone marrow CD34 cells.
Source: Journal of Allergy and Clinical Immunology - February 22, 2023 Category: Allergy & Immunology Authors: Alain Fischer, B énédicte Neven Tags: Editorial Source Type: research

An educational program for trainees in serving underserved primary immunodeficiency patients
Despite increasing awareness of barriers to healthcare, disparity of care in primary immune deficiency is a reality. Many of the clinical imperatives are larger in scope: political advocacy for newborn screening programs, establishing more centers of excellence in hematopoietic stem cell transplantation in impoverished areas, and combating systemic injustice. However, achieving these objectives depends on training a new generation of committed clinical immunologists.
Source: Journal of Allergy and Clinical Immunology - February 1, 2023 Category: Allergy & Immunology Authors: Jessica Galant-Swafford, Elizabeth George, Anna Meyer Source Type: research

Late Survival after Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease in a Single-Center Cohort
Chronic granulomatous disease (CGD) is an inborn error of immunity associated with significant morbidity and mortality for which hematopoietic stem cell transplant (HSCT) is a definitive treatment. In a large single-center cohort, we examined the late survival (survival at 5 years post-HSCT) of all patients who underwent HSCT for CGD at the Children ’s Hospital of Philadelphia (CHOP).
Source: Journal of Allergy and Clinical Immunology - February 1, 2023 Category: Allergy & Immunology Authors: Whitney Reid, Anne Wohlschlaeger, Brittany Adduce, Nancy Bunin, Jennifer Heimall Source Type: research

Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease
J Allergy Clin Immunol Pract. 2023 Jan 13:S2213-2198(23)00050-8. doi: 10.1016/j.jaip.2022.12.039. Online ahead of print.ABSTRACTChronic granulomatous disease (CGD) is a rare inborn error of immunity, resulting from a defect in nicotinamide adenine dinucleotide phosphate (NADPH) oxidation and decreased production of phagocyte reactive oxygen species. The main clinical manifestations are recurrent infections and chronic inflammatory disorders. Current approaches to management include antimicrobial prophylaxis and control of inflammatory complications. Hematopoietic stem cell transplantation (HSCT) or gene therapy can provide...
Source: Cancer Control - January 16, 2023 Category: Cancer & Oncology Authors: Alexander H Yang Brigit Sullivan Christa S Zerbe Suk See De Ravin Andrew M Blakely Martha M Quezado Beatriz E Marciano Jamie Marko Alexander Ling David E Kleiner John I Gallin Harry L Malech Steven M Holland Theo Heller Source Type: research

Specific Clinical and Immunological Changes Following Mesenchymal Stem Cell Transplantation in COVID-19-induced Acute Respiratory Distress Syndrome Patients: A Phase-I Clinical Trial
This study evaluated the safety and efficacy of three times transplantation of umbilical cord-derived MSCs (UC-MSCs) in terms of specific immunological and clinical changes in mild-to-moderate COVID-19-induced ARDS patients. In this single-center, open-label, phase 1 clinical trial, 20 patients diagnosed with COVID-19 and mild-to-moderate ARDS were included and were divided into two groups: a control group receiving standard care and an intervention group receiving UC-MSC in addition to standard care. Three consecutive intravenous transplants of UC-MSC (1× cells/kg body weight per each transplant) were performed in the in...
Source: Iranian Journal of Allergy, Asthma and Immunology - January 14, 2023 Category: Allergy & Immunology Authors: Najmeh Kaffash Farkhad Alireza Sedaghat Hamidreza Reihani Amir Adhami Moghadam Ahmad Bagheri Moghadam Nayereh Khadem Ghaebi Mohammad Ali Khodadoust Amin Reza Nikpoor Jalil Tavakol Afshari Source Type: research