Can I read Gene Therapy of Inherited Diseases on EtoBox?
Gene Therapy of Inherited Diseases by Alain Fischer; Marina Cavazzana-Calvo is a Social Sciences article available to read on EtoBox.
What is Gene Therapy of Inherited Diseases about?
alain.fi scher@nck. ap-hop-paris.fr Disease Effi cacy Toxic eff ects γ retrovirus (ex vivo) CD34 cells X-linked severe combined immunodefi ciency Yes, 9 years Yes, insertional mutagenesis Adenosine deaminase defi ciency Yes, 6 years No Chronic granulomatous disease Yes, transient Yes, insertional mutagenesis Fanconi's anaemia No No Skin stem cells Epidermolysis bullosa Yes, 10 months No Adeno-associated virus (in vivo) Muscle Haemophilia B Low level of expression No Liver Haemophilia B Yes, transient No Airway Cystic fi brosis No No Brain Ceroid lipofuscinosis No No Adenovirus (in vivo) Airway Cystic fi brosis No No Plasmids (liposomes; in vivo) Airway Cystic fi brosis No No Airway α antitrypsin defi ciency No No Brain Canavan disease No No Muscle Muscular dystrophy No No A more comprehensive list of clinical trials can be found in reference 2.
Who reads Gene Therapy of Inherited Diseases?
It is typically read by researchers, students, and practitioners in Social Sciences.
- Author
- Alain Fischer; Marina Cavazzana-Calvo
- Publisher
- Elsevier BV
- Published
- 2008
- Language
- EN
- Field
- Social Sciences