DNA gene therapy sequence

Genome

The DNA in the nucleus of a cell, containing around 20,000 protein coding genes.

RNA Polymerase

The enzyme that transcribes a gene's DNA into a complementary RNA transcript.

Introns

Segments of an RNA transcript that are removed during splicing and do not get translated into protein.

Exons

Segments of an RNA transcript that code for the final protein product and are joined during splicing to form mature mRNA.

Spliceosome

A large protein complex that edits the primary RNA transcript by removing introns and splicing exons together to produce mature mRNA.

mRNA

Messenger RNA, an intermediate copy of the information contained in DNA that is translated to protein.

Ribosome

A large protein-RNA complex in the cytoplasm that binds to mRNA and translates its sequence into a chain of polypeptides.

tRNA

Transfer RNA, a molecule specific to a particular amino acid that carries it to the ribosome during translation.

Polypeptide

A chain of amino acids formed during translation, whose sequence is determined by the nucleotide bases in DNA.

Protein Processing

The folding and assembly of the complete polypeptide chain into a 3D functional protein, occurring in the cytoplasm, endoplasmic reticulum, or Golgi apparatus.