The new lentiviral vector-based gene therapy works by inserting a functioning copy of the CFTR gene in the DNA of the epithelial cells in a patient’s airway ... “It’s been incredible to witness the ...

Researchers have identified the molecular and cellular mechanisms that transform healthy ... in stem cell maintenance and growth promotion) in combination with HPV oncogenes (genes that inhibit ...

How Healthy Stem Cells ... can help prevent blood stem cells from becoming malignant by removing certain receptors from their surfaces and blocking cancer gene activity, ... The Maternal ...

Now, researchers at University of California San Diego have discovered how healthy ... in stem cell maintenance and growth promotion) in combination with HPV oncogenes (genes that inhibit the ...

Cell and gene therapies (CGTs) represent the cutting edge of drug discovery. 1 These are amongst the most advanced medical treatments, specifically designed to correct the root genetic cause of an ...

Imagine a breakthrough in cancer treatment where only malignant cells are targeted, sparing healthy host cells; or patients with abnormal protein synthesis are treated to produce a healthy protein.

Traditionally, scientists have believed that cells adapt to changes and stressors in their environment through genetic mutations or by altering gene expression. But a new Yale-led study shows that ...

Although newborn screening includes CF, this typically only covers a selection of common genetic variants, and with over 2000 reported in the cystic fibrosis transmembrane conductance regulator (CFTR) ...