CRISPR-Cas9 corrects hypertrophic cardiomyopathy gene mutation in human preimplantation embryos [PreClinical]

1. The gene editing tool CRISPR-Cas9 was used to correct a mutant paternal MYBPC3 allele in human preimplantation embryos. 2. No off-target effects were detected. Evidence...

Small-molecule inhibitor may reduce chemotherapy-associated metastases [PreClinical]

1. In a mouse model of breast cancer, treatment with the chemotherapy paclitaxel (Ptx) enhanced tumor cell dissemination and the formation of micrometastatic lung...

Metformin treats Fragile X syndrome symptoms in mice [PreClinical]

1. Following treatment with metformin, mouse models of Fragile X syndrome (FXS) demonstrated restored neurological signaling activity and synaptic function. 2. Metformin administration reduced...

Combination treatment with calcineurin inhibitors and IL-2 enhances transplant survival [PreClinical]

1. Blood from liver transplant patients treated with a calcineurin inhibitor (CNI) showed increased levels of apoptotic regulatory T cells (Tregs) and lower IL-2...

Drug improves cystic fibrosis pulmonary inflammation and protein function [PreClinical]

1. Thymosin α1 (Tα1), an immunomodulatory drug currently in use, decreased lung inflammation in a mouse model of cystic fibrosis (CF). 2. In mice, administration...

Low-cost biochip isolates cells for clinical diagnosis [PreClinical]

1. Researchers manufactured a microfluidic biochip, using an inkjet printing system to apply conductive nanoparticles to a polymer substrate. 2. The biochip used an electric...

Lipid nanoparticles deliver messenger RNA for hemophilia B protein replacement therapy [PreClinical]

1. A lipid nanoparticle (LNP) delivery system was developed to introduce factor IX (FIX) messenger RNA (mRNA) into a FIX deficient mouse model of...

Next-generation metagenomics sequencing may sleuth out hard-to-find viruses in the blood

1. Next-generation metagenomics detected Human Hepegivirus-1 (HhpgV-1) in a cohort of patients who inject drugs. 2. The clinical significance of detecting HHpgB-1 infection is currently unclear. Evidence...

Novel imaging agent could enable pulmonary fibrosis diagnosis and monitoring [PreClinical]

  1. A gallium-labeled, collagen-binding peptide (68Ga-CBP8) specifically identified fibrotic tissue via positron emission tomography (PET) imaging in two mouse models of idiopathic pulmonary fibrosis...

Glucocorticoid compounds improve stem cell transplant efficiency in mice [PreClinical]

  1. Following treatment with glucocorticoids, human hematopoietic stem cells (HSCs) from cord blood demonstrated enhanced homing and engraftment to bone marrow in mice. 2. Glucocorticoids...
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