Your privacy, your choice

We use essential cookies to make sure the site can function. We also use optional cookies for advertising, personalisation of content, usage analysis, and social media.

By accepting optional cookies, you consent to the processing of your personal data - including transfers to third parties. Some third parties are outside of the European Economic Area, with varying standards of data protection.

See our privacy policy for more information on the use of your personal data.

for further information and to change your choices.
Skip to main content
Fig. 2 | Journal of Biomedical Science

Fig. 2

From: Gene therapy for ultrarare diseases: a geneticist’s perspective

Fig. 2

Mechanisms of lentiviral vector-modified autologous CD34+ hematopoietic stem cell transplantation. Transduction of hematopoietic stem cells can correct functional defects in blood cells, including T cells, B cells, and macrophages. Transgenes can encode secretory proteins that are secreted into the systemic circulation. The secreted protein, if it contains a brain-targeting epitope, can enter the brain. Bone marrow-derived cells can also migrate to the brain and differentiate into microglia, which can ameliorate brain defects

Back to article page

Journal of Biomedical Science

ISSN: 1423-0127