Welcome to Gene Therapy Science / Learning Center
Welcome to the Hemophilia Gene Therapy Learning Center. Here you will find links to further resources to explore the topics on this website in more detail.
The Hemophilia Gene Therapy Webinar Series will explore the complex science underpinning hemophilia gene therapy. Hosted by an expert hematologist, joined by a specialist, each webinar will focus on providing a high-science review of key areas of interest in hemophilia gene therapy.
Gerry Dolan and Thierry VandenDriessche explore the complex processes involved in gene therapy, from transgene packaging to expression of the protein of interest.
Andreas Tiede and Heiner Wedemeyer explore the role of the liver in hemophilia gene therapy, including its physiology, its tolerogenic nature, and key considerations for liver-targeted gene therapy.
Lindsey George and Jude Samulski investigate key characteristics of adeno-associated virus as a platform to produce recombinant vectors for gene therapy, including current understanding of its dynamics and function.
The Hemophilia Gene Therapy Brochures are complementary to the webinar series. Each brochure delves deeper into the topic covered during the webinar episode. These can be viewed online or downloaded using the links below.
The following videos provide additional educational content around the key concepts important for understanding hemophilia gene therapy.
3' refers to the end of a single-stranded nucleic acid chain to which a hydroxyl group (-OH) is attached to the 3'-carbon atom of the nucleotide. 1
5' refers to the end of a single-stranded nucleic acid chain to which a phosphate is attached to the 5'-carbon atom of the nucleotide.1
The capsid is the protein shell of a virus that protects the genetic material while interacting with the host environment.2 Capsid proteins determine cell-type specificity.3
A chromosome is an organized package of DNA found in the nucleus of a cell. Humans have 23 pairs of chromosomes–22 pairs of numbered chromosomes, called autosomes, and one pair of sex chromosomes, X and Y.4
A codon is a sequence of three nucleotides that codes a specific amino acid. For DNA, there are four different nucleotides (A, T, C, or G) from which a codon can be composed.5
A cellular process by which substances are brought into a cell. The substance is surrounded by an area of cell membrane, which then buds off inside the cell to form a vesicle containing the ingested material.6
An enhancer is an upstream regulatory DNA sequence that provides binding sites to regulatory proteins and can augment the activity of a promoter.7
Exogenous DNA that remains physically independent of the cell’s endogenous chromosome or complement of chromosomes.8
Harvesting and cultivating of patient cells in the laboratory. Cells are incubated with vectors carrying a corrective or therapeutic gene. Cells with the new genetic information are then transplanted back into the patient from whom they were derived.9
Removal, disruption or correction of faulty elements of DNA within the gene.10
Addition of a functional copy of a missing gene or augmentation of a gene that is non-functional into target cells to produce more of a protein.10,11
The ability of a substance, such as an antigen or epitope, to trigger an immune response in the host.12
An intron is a portion of DNA that does not code for an amino acid.13
ITRs are 145-bp sequences that frame the expression cassette.14
Administration of a vector carrying the therapeutic genetic material to a live animal. The vector can be delivered by a variety of methods, including direct injection into the blood (intravenous injection) or by various organs by other physical means of administration (hypodermic injection, aerosol, intrathecal, etc.).9
Single molecule of RNA that works as a chemical map for a protein product.9
Membrane-bound organelle that contains the cell's chromosomes. Pores in the nuclear membrane allow for the passage of molecules in and out of the nucleus.15
An extrachromosomal, self-replicating piece of DNA. Plasmids are usually circular and transferable between cells.16
Sequence of DNA, typically at the 5’ region, where regulatory elements such as transcription factors bind and initiate transcription of the associated gene.17
A large protease complex which selectively degrades proteins by proteolysis. The proteasome works in collaboration with ubiquitin–polymerization of ubiquitin serves as a degradation signal that transports the target proteins to the proteasome for degradation.18
The proportions of individuals within a population with an antibody to a serotype. Seroprevalence is measured in blood serum.19
Group of closely related microorganisms distinguished by a characteristic set of antigens and detected by an antibody.9
The Poly(A) signal sequence acts as the transcription terminator, halting transcription once the transgene is fully transcribed.20
Transfer of genetic material into the nucleus of a cell, such that elements of the newly transferred DNA are then expressed. This can be accomplished naturally by a virus or other vector or experimentally by augmenting the receptivity of the cell membrane of the recipient cell with chemicals or electricity.21
tRNAs act as adaptors between the mRNA and the amino acids during translation. The tRNA has an anticodon loop that binds to the complementary mRNA codon and also has a bound amino acid.22
The transgene is the nucleic acid sequence encoding an artificially added gene.23
The ability of a virus to infect a particular type of cell in the body.23
A gene therapy delivery vehicle, which encapsulates a therapeutic gene and delivers it to target cells. Vectors can be either virus-derived or non-viral.9
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