The following info has been kindly provided by Juan Carlos Baiges This publication shows the CNS gene therapy results (in mouse) with different viruses than the herpes viruses. Interestingly, in this case there is no integration in the genome which minimizes the risk of insertional mutagenesis, which is a known problem in gene therapy and that is causing more delays in the development of these therapies. Greetings Juan Carlos Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors A Rahim1,2,3, A M S Wong4, S J Howe2, S M K Buckley3, A D Acosta-Saltos1, K E Elston4, N J Ward2, N J Philpott2, J D Cooper4, P N Anderson5, S N Waddington3, A J Thrasher2,6 and G Raivich1,5 1Perinatal Brain Protection and Repair Group, Department of Obstetrics and Gynaecology, University College London, London, UK 2Molecular Immunology Unit, Centre for Immunodeficiency, Institute of Child Health, University College London, London, UK 3Department of Haematology, Haemophilia Centre and Haemostasis Unit, Royal Free and University College Medical School, London, UK 4Pediatric Storage Disorders Laboratory, Department of Neuroscience, Institute of Psychiatry, Kings College London, London, UK 5Department of Anatomy and Developmental Biology, University College London, London, UK 6Great Ormond Street Hospital NHS Trust, London, UK Correspondence: Dr AJ Thrasher, Molecular Immunology Unit, Centre for Immunodeficiency, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK. E-mail: This email address is being protected from spambots. You need JavaScript enabled to view it. Received 12 June 2008; Revised 2 December 2008; Accepted 3 December 2008; Published online 22 January 2009. Abstract Non-integrating lentiviral vectors show considerable promise for gene therapy applications as they persist as long-term episomes in non-dividing cells and diminish risks of insertional mutagenesis. In this study, non-integrating lentiviral vectors were evaluated for their use in the adult and fetal central nervous system of rodents. Vectors differentially pseudotyped with vesicular stomatitis virus, rabies and baculoviral envelope proteins allowed targeting of varied cell populations. Efficient gene delivery to discrete areas of the brain and spinal cord was observed following stereotactic administration. Furthermore, after direct in utero administration (E14), sustained and strong expression was observed 4 months into adulthood. Quantification of transduction and viral copy number was comparable when using non-integrating lentivirus and conventional integrating vector. These data support the use of non-integrating lentiviral vectors as an effective alternative to their integrating counterparts in gene therapy applications, and highlight their potential for treatment of inherited and acquired neurological disorders.

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!


Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.



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