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  2. Gene therapy - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy

    Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological ...

  3. Site-directed mutagenesis - Wikipedia

    en.wikipedia.org/wiki/Site-directed_mutagenesis

    Site-directed mutagenesis is a molecular biology method that is used to make specific and intentional mutating changes to the DNA sequence of a gene and any gene products. Also called site-specific mutagenesis or oligonucleotide-directed mutagenesis , it is used for investigating the structure and biological activity of DNA , RNA , and protein ...

  4. Mutagenesis (molecular biology technique) - Wikipedia

    en.wikipedia.org/wiki/Mutagenesis_(molecular...

    Types of mutations that can be introduced by random, site-directed, combinatorial, or insertional mutagenesis. In molecular biology, mutagenesis is an important laboratory technique whereby DNA mutations are deliberately engineered to produce libraries of mutant genes, proteins, strains of bacteria, or other genetically modified organisms.

  5. FDA approves Pfizer's first gene therapy for rare inherited ...

    www.aol.com/news/fda-approves-pfizer-first-gene...

    The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...

  6. Therapeutic gene modulation - Wikipedia

    en.wikipedia.org/wiki/Therapeutic_gene_modulation

    Therapeutic gene modulation refers to the practice of altering the expression of a gene at one of various stages, with a view to alleviate some form of ailment. It differs from gene therapy in that gene modulation seeks to alter the expression of an endogenous gene (perhaps through the introduction of a gene encoding a novel modulatory protein) whereas gene therapy concerns the introduction of ...

  7. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Gene therapies. Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase ... "Gene Therapy Arrives". Scientific American This page was last ...

  8. Modifications (genetics) - Wikipedia

    en.wikipedia.org/wiki/Modifications_(genetics)

    Genetic modifications can occur naturally, through aforementioned mutations in an organism's genome, or through biotechnological methods of selecting a gene of interest to manipulate in order to make something new or improve upon what already exists. [1]

  9. What happens when a $2 million gene therapy is not enough - AOL

    www.aol.com/news/happens-2-million-gene-therapy...

    Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak ...