W10

• Gene therapies


  Processes that use introduction/alteration of DNA sequences to create a therapeutic benefit
• Regenerative medicine


  Medicine that seeks to restore tissue structure and function and provide options for what were previously untreatable injuries or diseases
• Cell therapy


  Transfer of live cells that have often been modified or grown outside of the body for gene therapy, and/or tissue bioengineering
• Sickle cell disease


  Recessive inherited blood disorder that effects hemoglobin and is caused by an SNP
• Casgevy


  The first ever FDA approved CRISPR treatment that was approved for the treatment of sickle cell anemia
• iPSCs


  Induced pluripotent stem cells that are made to be pluripotent in a lab
• Yamanaka factors


  Transcription factors that are master regulators used to create iPSC
• Master regulators


  Transcription factors that can make cells pluripotent
• Molecular chaperones


  Molecules that bind and release proteins repeatedly to to help them fold into the most stable form
• Hydrophobic regions


  The parts of proteins can be bound by molecular chaperones to prevent them from aggregating until the protein has folded correctly
• Heat shock proteins


  Group of molecular chaperones that create folding chambers for proteins
• Paracrine


  Cell signaling type that involves secreted factors limited to a local range
• Juxtacrine


  Cell signaling that is mediated by contact and not secrete diffusible factors
• Autocrine


  A type of cell signaling that occurs when a cell produces signaling factors that affect itself
• Nuclear receptors


  Usually transcription factors. Ligands binding to them results in a change in control of transcription
• Integrins


  Transmembrane proteins that help cells adhere to the extracellular matrix
• Anoikis


  A term for apoptosis that occurs in response to a loss of adhesion to other cells. Aka death due to lack of integrin signaling
• Morphogens


  Paracrine factors that influence cell fate
• GTPases


  Proteins that are turned on when bound to GTP and turned off when bound to GDP
• GAPs


  Proteins that promote turning GTPases off
• GEFs


  Proteins that promote turning GTPases on
• Kinases


  Proteins that add phosphates form ATP to particular amino acids
• Phosphatases


  Proteins that remove phosphate groups from particular amino acids