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Clinical Implications of Basic Research
Stem Cells and Spinal Cord Repair
For the past couple of decades, clinicians have watched the stem-cell field with a mixture of anticipation and skepticism. No group of patients has been more expectant than those with spinal cord injuries. Therapies for spinal cord injury have been promised almost since the dawning of the stem-cell…
Clinical Implications of Basic Research
Gene Therapy Meets Stem Cells
Stem cell–based therapies have the potential to repair and even correct the defects related to human diseases. Although tantalizing niche applications have moved forward in the clinical setting, progress seems to be slow, and ethical challenges have yet to be definitively addressed. The goal of…
Perspective
Selling Bone Marrow — Flynn v. Holder
On December 1, 2011, in Flynn v. Holder, the U.S. Court of Appeals for the Ninth Circuit held that the ban on selling "bone marrow" that is part of the National Organ Transplant Act (NOTA) of 1984 does not encompass "peripheral blood stem cells" obtained through apheresis. This ruling means that…
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Clinical Implications of Basic Research
Diabetic Stem-Cell “Mobilopathy”
Hematopoietic stem-cell (HSC) transplantation remains the primary curative treatment for patients with a variety of hematologic cancers. Transplantation of either autologous or allogeneic stem cells requires the acquisition of sufficient numbers of HSCs to ensure rapid and consistent trilineage…
Editorial
The Yin and Yang of Interleukin-2–Mediated Immunotherapy
In this issue of the Journal, the findings of two case series suggest that in vivo treatment with interleukin-2 can suppress immune-mediated diseases. In one study, Koreth et al. found that low-dose interleukin-2 was associated with reversal of glucocorticoid-refractory chronic graft-versus-host…
Perspective
The Public, Political Parties, and Stem-Cell Research
Until recently, it seemed as if the question of future federal funding for embryonic stem-cell research had been settled. The Obama administration had lifted President George W. Bush's previous restrictions on funding, and the legality of that decision was upheld by federal courts. But the 2012…
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The 2012 presidential election campaign has created uncertainty about federal funding for human embryonic stem-cell research, with most Republican candidates suggesting that they would substantially reduce such funding. Where do U.S. voters stand on this issue? This Perspective summarizes polling results.
Editorial
Eliminating Cells Gone Astray
The therapeutic use of cells from healthy donors or patients is increasing. Decades ago, transfusion medicine and bone marrow transplantation provided the first successful cell therapeutics and established the foundations for cell delivery. Clinical investigation soon uncovered the double-edged…
Original Article
Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy
Although cellular therapies may be effective in cancer treatment, their potential for expansion, damage of normal organs,– and malignant transformation is a source of concern. In contrast, the toxic effects of small molecules usually diminish once the drugs are withdrawn. One approach to…
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Original Article
Cord Colitis Syndrome in Cord-Blood Stem-Cell Transplantation
Hematopoietic stem-cell transplantation (HSCT) with the use of umbilical-cord blood is effective in patients for whom a sibling or matched, unrelated donor is not available.– Acute graft-versus-host disease (GVHD), a major cause of illness in patients undergoing HSCT, is less severe with cord…
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Clinical Implications of Basic Research
Stem Cells and Eye Development
Stem cells hold promise in the treatment of many different types of disease. The generation of induced pluripotent stem cells (iPSCs) by reprogramming somatic cells has provided unprecedented opportunities for studying basic biology and modeling human diseases. In addition, the differentiation of…
Perspective
Human Embryonic Stem-Cell Research under Siege — Battle Won but Not the War
The struggle over federal funding for research involving human embryonic stem cells (ESCs) continues — and may well be waged for years to come. On August 23, 2010, Chief Judge Royce Lamberth of the U.S. District Court of the District of Columbia granted the motion of plaintiffs James Sherley and…
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Clinical Implications of Basic Research
Induced Pluripotent Stem Cells — A Cautionary Note
In 2006, when Shinya Yamanaka found that introducing just four genes into normal mouse skin cells made those cells pluripotent — and thus able to generate all cell types of the body — the reaction was almost one of disbelief. But less than a year later, both he and James Thomson did the same…
Perspective
Sudden Death for a Challenge to Federal Funding of Stem-Cell Research
The National Institutes of Health (NIH) and researchers using human embryonic stem cells (ESCs) have been operating under some uncertainty since the August 2010 ruling by federal districtcourt judge Royce Lamberth that the Dickey–Wicker Amendment (which Congress has attached to all appropriations…
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Original Article
Evidence for Human Lung Stem Cells
The fundamental properties of stem cells are self-renewal, clonogenicity, and multipotentiality. Although tissue-specific stem cells have been identified,, they have not always been shown to exhibit stem-cell properties, particularly in the mammalian lung. Various cell populations with some of…
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Editorial
Toward Lung Regeneration
During the fifth gestational week, the human lung begins to develop from epithelial buds of a primordial endodermal tube that is destined to give rise not only to the lung but also to most abdominal organs. Using tools to trace individual cells, biologists have determined that virtually all of the…
Clinical Implications of Basic Research
In Utero Hematopoietic Stem-Cell Transplantation — A Match for Mom
Once researchers recognized that adult stem cells can generate multiple cell types and contribute to tissue homeostasis, it became conceivable to exploit this potential to treat genetic or acquired disorders characterized by tissue degeneration or organ dysfunction. The concept of regenerative…
Review Article
Medical Progress: Multiple Myeloma
Multiple myeloma is a neoplastic plasma-cell disorder that is characterized by clonal proliferation of malignant plasma cells in the bone marrow microenvironment, monoclonal protein in the blood or urine, and associated organ dysfunction. It accounts for approximately 1% of neoplastic diseases and…
- CME
Editorial
The Role of Allogeneic-Cell Transplantation in Leukemia
In the early 1970s, patients with high-risk hematologic cancers had a small chance of long-term survival. This chance began to increase with allogeneic blood or bone marrow transplantation, which combines high-dose chemotherapy, total-body irradiation, and graft-versus-leukemia effects. In…
Original Article
Brief Report: Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome
The Wiskott–Aldrich syndrome (WAS) is a complex primary immunodeficiency disorder that is characterized by recurrent infections, thrombocytopenia, eczema, and autoimmunity, and caused by mutations in WAS. Its gene product, WAS protein (WASP), is a key regulator of actin polymerization in…
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Perspective
Resurrection of a Stem-Cell Funding Barrier — Dickey–Wicker in Court
Embryo research was born political. Expressions of shock and surprise at the August 23 ruling of federal district court judge Royce Lamberth enjoining federal funding of stem-cell research — which was based largely on his reading of an amendment to an appropriations bill — are thus not terribly…
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