Fast Facts: Gene Therapy
R. Herzog, L. Popplewell
- 104 pages
- English
- ePUB (mobile friendly)
- Available on iOS & Android
Fast Facts: Gene Therapy
R. Herzog, L. Popplewell
About This Book
Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: ā¢ Principles of gene therapy ā¢ Gene therapy techniques ā¢ Ethical and safety considerations ā¢ Gene therapies with proven clinical efficacy ā¢ Genome editing ā¢ Research directions ā the next wave of treatments
Frequently asked questions
Information
4 | Gene therapies with proven clinical efficacy |
Hereditary blindness
TABLE 4.1 |
Approved gene therapy medications* |
Yescarta (axicabtagene ciloleucel; FDA, EMA): CAR-T cell therapy directed against CD19 in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy Kymriah (tisagenlecleucel; FDA, EMA): CAR-T cell therapy directed against CD19 in patients up to 25 years of age with B-cell acute lymphoblastic leukemia that is refractory or in second or later relapse; and in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy Luxturna (voretigene neparvovec; FDA, EMA): AAV vector for the treatment of Leber congenital amaurosis (by subretinal injection) Zolgensma (onasemnogene abeparvovec-xioi; FDA): AAV vector for the treatment of patients younger than 2 years of age with spinal muscular atrophy (by systemic delivery) Imlygic (talimogene laherparepvec; FDA, EMA): oncolytic viral therapy based on herpes simplex virus expressing GM-CSF for the treatment of unresectable lesions in patients with melanoma recurrent after initial surgery Strimvelis (EMA): retroviral vector for the treatment of ADAāSCID by HSC gene therapy |
*At the time of writing. AAV, adeno-associated virus; ADAāSCID, adenosine deaminase deficiencyāsevere combined immunodeficiency; CAR, chimeric antigen receptor; DLBCL, diffuse large B-cell lymphoma; EMA, European Medicines Agency; FDA, (US) Food and Drug Administration; GM-CSF, granulocyte-macrophage colony-stimulating factor; HSC, hematopoietic stem cell. |
Disease | Vector | Site of gene transfer | Route of administration | Target cells/tissue | Transgene product |
LCA | AAV2 | In vivo | Subretinal | Retinal epithelial cells | RPE65 |
SMA | AAV9 | In vivo | Intravenous | Motor neurons | SMN1 |
MLD | Lentivirus | Ex vivo to autologous HSC | HSC transplant | CNS | ARSA |
ADAāSCID | Retrovirus | Ex vivo to autologous HSC | HSC transplant | Immune system | ADA |
Hemophilia A | AAV (different capsids) | In vivo | Intravenous | Hepatocytes | Factor VIII |
Hemophilia B | AAV (different capsids) | In vivo | Intravenous | Hepatocytes | Factor IX |
B-ALL | Lentivirus | Ex vivo to primary T cells | T-cell transplant | CD19-expressing cells | CD19-specific CAR |
DLBCL | Lentivirus | Ex vivo to primary T cells | T-cell transplant | CD19-expressing cells | CD19-specific CAR |
AAV, adeno-associated virus; ADA, adenosine deaminase; ADAāSCID, ADA deficiencyāsevere combined immunodeficiency; ARSA, arylsulfatase A; B-ALL, B-cell acute lymphoblastic leukemia; CAR, chimeric antigen receptor; CNS, central nervous system; DLBCL, diffuse large B-cell lymphoma; HSC, hematopoietic stem cell; LCA, Leber congenital amaurosis; MLD, metachromatic leukodystrophy; RPE65, retinal pigment epithelium 65 kDa protein; SMA, spinal muscular atrophy; SMN1, survival of motor neuron 1, telomeric. |