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Combined stem cell-gene therapy approach seen as potential treatment for cystic fibrosis

A new study has provided proof of principle for a combined stem cell-gene therapy approach as a potential treatment for cystic fibrosis. The researchers were able to correct the CFTR gene defect using adult stem cells and ex vivo gene therapy, which may offer advantages in screening and long-term function.

SourceUniversity of Pittsburgh Medical Center·JournalProceedings of the National Academy of Sciences·DateDec 20, 2004

Stem cells to the rescue - or not?

Researchers transplanted BM-SP stem cells into mice with cardiomyopathy, but found that only 2 muscle fibers expressed restored sarcoglycan levels. The study suggests alternative approaches should be investigated for regenerative medicine.

SourceJCI Journals·JournalJournal of Clinical Investigation·DateDec 1, 2004

New gene mutation found to cause 'bubble boy disease'

Researchers identified a complete deficiency in the CD3 epsilon chain of the T cell receptor causing SCID, leading to normal B cells but no T cells. Early diagnosis via bone marrow stem cell transplantation can result in a survival rate as high as 97% for patients with this mutation.

SourceJCI Journals·JournalJournal of Clinical Investigation·DateNov 15, 2004

NIH launches study of hematopoietic stem cell transplantation for severe, treatment-resistant lupus

Researchers at the NIH will conduct a clinical study to assess the safety and efficacy of hematopoietic stem cell transplantation in treating severe forms of lupus. The study aims to create a new immune system that doesn't attack healthy cells, offering hope for patients with limited treatment options.

Origin of multiple myeloma found in rare stem cell

Researchers at Johns Hopkins Medicine discovered a rare stem cell in multiple myeloma that gives rise to malignant bone marrow plasma cells. Current treatments may not be effective against this errant stem cell, leading scientists to explore dual-therapy approaches to target both the visible and root causes of the disease.

SourceJohns Hopkins Medicine·JournalBlood·DateDec 3, 2003

IV infustions of human umbilical cord blood stem cells benefit rodents with ALS, spinal cord injury

Researchers found that intravenous infusions of human umbilical cord blood stem cells delayed ALS disease progression by at least two to three weeks and improved survival in mice. The cells also circulated to organs outside the central nervous system, suggesting an immune-protective mechanism.

SourceUniversity of South Florida (USF Health)·JournalJournal of Hematotherapy & Stem Cell Research·DateJun 12, 2003

Cancer therapy may offer lupus patients new hope

Researchers at Johns Hopkins Lupus Center and Kimmel Cancer Center found that high-dose intravenous cyclophosphamide can lead to complete responses and partial responses in lupus patients who failed standard therapy. The treatment also had fewer side effects compared to traditional treatments.

SourceJohns Hopkins Medicine·JournalArthritis & Rheumatism·DateJan 10, 2003

The Lancet Neurology launched

The Lancet Neurology aims to report the most important advances in neurology, predicting risk of neurological conditions through genetics and imaging. Experimental therapies like gene therapy and stem cells offer potential for treating neurological disorders.

SourceThe Lancet_DELETED·JournalThe Lancet Neurology·DateApr 15, 2002

Carolina scientists the first to identify and purify liver stem cells

Carolina scientists have identified and purified hepatic stem cells, which can regenerate liver and bile duct tissue. The accomplishment marks a milestone for future liver regeneration through cellular therapy, potentially reducing the need for whole-organ transplants in patients with various liver diseases.

SourceUniversity of North Carolina Health Care·JournalProceedings of the National Academy of Sciences·DateNov 13, 2000

Stem cells may be powerful gene shuttle

Researchers from HHMI at Children's Hospital and Harvard Medical School discovered that stem cells can deliver therapeutic genes to diseased organs, potentially remodeling genetically defective tissues. The study suggests that adult stem cells may be manipulated to heal genetic defects in various organs and tissues.

Enzyme-based method to isolate stem cells from umbilical cord blood shows promise

Researchers have developed a new method to identify and isolate stem cells from umbilical cord blood using an enzyme that changes a fluorescent tag. This technique could help investigate fundamental questions about stem cells and potentially improve success rates of stem cell transplants by eliminating unwanted mature blood cells.

SourceDuke University Medical Center·JournalProceedings of the National Academy of Sciences·DateAug 2, 1999