Novel therapeutic strategy for single gene disorders delivers RNA that encodes the missing protein

(Mary Ann Liebert, Inc./Genetic Engineering News) Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin -- which is missing or nonfunctional in the genetic disorder AAT deficiency -- into cells in the laboratory, enabling the cells to produce highly functional AAT. This innovative approach to treating single gene disorders such as AAT deficiency offers and safe, simpler, and more cost-effective alternative to gene therapy or protein replacement, according to the authors of the study published in Nucleic Acid Therapeutics.
Source: EurekAlert! - Medicine and Health - Category: Global & Universal Source Type: news

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Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
TH and MW contributed equally to this work.BackgroundMultiple myeloma is caused by an accumulation of malignant plasma cells in the bone marrow. Myeloma is characterized by an osteolytic bone disease, caused by increased bone degradation and reduced bone formation. Bone morphogenetic proteins (BMPs) are members of the transforming growth factor (TGF)-β superfamily. BMP-signaling is important for both pre- and postnatal bone formation. Additionally, several BMPs induce growth arrest and apoptosis in myeloma cells. Thus, increasing BMP-signaling in myeloma patients may reduce tumor growth and restore bone formation. We ...
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Source: The Yale Journal of Biology and Medicine - Category: Universities & Medical Training Tags: Yale J Biol Med Source Type: research
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