Demystify Your Disease’s R&D Readiness

Genetic Therapies

Gene therapy, genome editing, RNA-based therapy, and cell therapy are several of the promising therapies making headlines in the popular press. These advanced biologic products have the potential to treat, and in some cases cure, many types of rare diseases and cancer. However, it can be hard to know if any have the possibility to treat your disease. You may wish to gain a basic understanding of these intriguing advances and then discuss their relevance to your disease with your Medical/Scientific Advisory Board and possibly experts in the respective therapy field.

  • Gene therapy is a technique that modifies a person’s genetic material (DNA or RNA) to treat or cure a disease.
  • CRISPR-Cas gene editing system (CRIPSR) is just one type of genome editing, but is currently regarded as an editing technique that may be able to be treat or cure human genetic disorders
    • CRISPR-Cas gene editing system is composed of a short strand of RNA, often called guide RNA (gRNA), that is made to target a specific sequence of DNA. The gRNA strand is surrounded by short repeated sequences of RNA that act as scaffolding. The system is linked to an enzyme that is capable of cutting double-stranded DNA.
    • Currently CRISPR is most commonly used to make disease cell and animal models, but is also being explored as to perform rapid diagnosis and to correct disease causing genetic changes.
    • There are different CRISPR-Cas gene editing systems, including CRISPR-Cas9.
    • CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.
    • Cas stands for CRISPR associated protein.
    • Learn more about genome editing and CRISPR-Cas9:
      • What is Genome Editing? is a short webpage by the the National Human Genome Research Institute (NHGRI) introducing CRISPR and also provides links to more in depth CRISPR topics at the bottom of the page. 
      • What are genome editing and CRISPR-Cas9? is a short webpage by MedlinePlus (from the National Library of Medicine) introduces genome editing in plain language and also provides links to other resources including published scientific journal articles focused on the topics.
      • What is CRISPR-Cas9? is a webpage by Wellcome Genome Campus YourGenome that provides basic information about CRISPR-Cas9 in easy to read bullet points and includes diagrams depicting the steps of genome editing.
  • Chimeric Antigen Receptor T-Cells (CAR T-Cells) is a type of treatment in which a patient’s T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells.
    • CAR T-Cells are made by adding a gene for a special receptor that binds to a certain protein on the patient’s cancer cells to the patient’s T cells harvested from their own blood. The special receptor is called a chimeric antigen receptor (CAR).
    • Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion, enabling the a patient’s immune cells to fight their own cancer.
    • CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers is a webpage by the National Cancer Institute that provides an overview of the development of the CAR T-cell therapy as well as its expanding its use.

Resources

Research Tools
Genetic Therapies
Finding Disease and Technology Info
Keeping Up with Advances