Glossary

C

Chimeric: Composed of two or more parts of different origin. Example would be an artificial protein composed of two parts of different natural proteins.

Directed evolution: Approach to protein design in which protein variants with desired properties are selected from a large library of variants. Variants emerging from the selection are then randomly mutated, and the selection/mutation process is iterated until the desired properties are optimized.

ELISA: Enzyme-Linked ImmunoSorbent Assay. Method for assaying the amount of a target protein by binding antibodies to it that are linked to an enzyme capable of generating a colored end product. Thus, the more antibody-linked enzyme that is present, the faster and higher the colored end product is produced.

FACS: Fluorescence-activated cell sorting. A specialized flow-cytometry technique used to sort a heterogeneous mixture of biological cells into two or more populations, based upon the fluorescent characteristics of each cell.

Gene repair: An approach to treatment of disease which attempts to restore target cell function by repairing damaged genes in a target cell's genome.
Gene therapy: An approach to treatment of disease which attempts to alter or restore target cell function by adding new genes to the target cell's genome.
Genome engineering: An emerging scientific discipline whose goal is the development of tools and methods for precise manipulation of organism genomes.

Hematopoietic stem cells: Stem cells from which all blood cell types are derived.
Heterodimeric: Composed of two non-identical subunits.
Homing endonucleases: Rare-cutting enzymes most often encoded by introns or inteins that promote the spread and persistence of their host elements by specifically inducing double strand breaks in homologous alleles lacking intervening sequences. Homing endonucleases balance long target sites of 14-40 base pairs with tolerance for point mutations at selected positions to arrive at their high specificity of around 1x109, making them essentially gene-specific.

Immune reconstitution: The degree of immune function achieved after new bone marrow stem cells are engrafted into a patient. When new bone marrow stem cells are engrafted into a patient, these stem cells must produce new immune cells which merge into a properly functioning immune system. How well the stem cells are able to do this determines the degree of immune reconstitution achieved.
Immunodeficiency disorders: Diseases which affect immune function. They may be primary, in which case they are inherited defects in specific genes involved in immune function, or may occur secondary to viral infections such as HIV.
Isothermal titration calorimetry (ITC): A quantitative biophysical technique used to determine thermodynamic properties of biochemical interactions.

LAGLIDADG homing endonucleases (LHEs): A class of homing endonucleases distinguished by the possession of either one or two copies of the conserved 10-residue LAGLIDADG sequence motif.
Lymphopoiesis: Development of lymphoid cells from bone marrow precursors.

MACS: Magnetic cell separation. Selected cells in a heterogeneous population are bound to magnetic beads based on their affinity for molecules on the bead surface. The mixture is run through a magnetized column which retains bead-bound cells, separating them from non-bound cells which flow through.

Noncognate: Not evolutionarily related.
Non-integrating lentiviral vectors (NIL): A class of virus used to deliver genes to cells that will not integrate into the DNA of the infected cell.
Non-myeloablative: Approach to bone marrow transplantation in which doses of chemotherapy given prior to infusion of new stem cells are of sufficient magnitude to suppress but not entirely ablate host bone marrow hematopoiesis. The intent is to sufficiently suppress host bone marrow function that the new stem cells can efficiently engraft, without so impairing host bone marrow function that the patient becomes markedly susceptible to infection.
Non-NRSA: Type of NIH training grant for researchers who do not qualify for NRSA grants. Foreign citizens with appropriate work authorization are eligible for these grants.
NRSA: Ruth L. Kirschstein National Research Service Award. Type of NIH training grant to US citizens, nationals or permanent residents.

PSSM analysis: Position specific search matrices. Method for identifying target sites in large DNA fragments based on properties of the target sites contained in user-defined matrices.

Rosetta Design: A computer program developed by David Baker’s laboratory at the University of Washington for the design and optimization of protein-protein and protein-DNA interfaces.

Somatic hypermutation: Method utilized by the body to mutate antibody proteins to generate diverse antibody libraries from which high affinity antibodies can be selected. An important component of the body's approach to directed evolution of antibodies during an immune response.

X-linked immunodeficiency (XID): A murine immune deficiency caused by mutations in the protein Bruton's Tyrosine Kinase. XID is a model for the human disease X-linked agammaglobulinemia which is caused by mutations in the same protein.
X-linked severe combined immunodeficiency (XSCID): Human immune deficiency caused by mutations in the cytokine receptor common gamma chain, a component of multiple cytokine receptors involved in regulating lymphopiesis and immune function. Loss of function mutations in the common gamma chain cause an early failure of T-cell and NK cell development, resulting in severe immune deficient. Commonly referred to as "Bubble boy syndrome."