Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
نویسندگان
چکیده
منابع مشابه
Hematopoietic stem cell gene therapy.
Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clini...
متن کاملHematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD progression can be halted by allogeneic hematopoietic cell transplantation (HCT). We initiated a gene therapy trial in two ALD patients for whom there were no matched donors. Autol...
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Thalassemia is an autosomal recessive disorder associated with defective synthesis of the α- or β-chain of hemoglobin. For β-thalassemia major patients, therapeutic options are either monthly red cell transfusions and chelation therapy or allogeneic stem cell transplant. Stem cell transplant is the only curative approach and success is inversely correlated with the degree of iron overload and h...
متن کاملHematopoietic stem cell gene replacement therapy.
Human gene therapy trials have begun, and by early reports are a success [1]. The initial clinical trial which demonstrated the safety of infusing genetically marked lymphocytes into humans was reported in 1990 [2]. This paved the way for researchers at the National Institutes of Health (NIH) :o begin the first therapeutic trials of treating adenosine deaminase deficient patients with genetical...
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The introduction and evolution of hematopoietic stem cell transplantation (HSCT) could be traced back to 1950s, to the studies on interactions among irradiation, covering spleen and bone marrow from it and injection of bone marrow cells. Today, HSCT is considered a well-established, effective and promising means of therapy for various malignant and non-malignant medical conditions, both in chil...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2017
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa1700554