Gene Therapy for SCID-X1 Using a Self Lentiviral Vector

2017-07-17 09:53:50 | BioPortfolio


This is a Phase I/II clinical trial of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a self lentiviral vector to replace the defective genes with good genes. The primary objectives are to evaluate the safety and efficacy of the ex vivo gene therapy clinical protocol.


X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder caused by defects in the common cytokine receptor chain, normally on the surface of lymphocytes. Individuals with SCID-X1 fail in the development of the immune system and so have difficulty fighting infections, which may lead to chronic or severe illness and death, unless they are rescued by a bone marrow transplant from a healthy donor. This trial aims to treat SCID-X1 using a self lentiviral vector to replace the defective genes with good genes. By collecting an individual's stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned into the blood to help produce normal healthy immune cells.

The primary objectives are to evaluate the safety of the ex vivo gene therapy clinical protocol and the efficacy of immune reconstitution allowing the cure of infections present at the time of gene therapy, assessment of integration sites, and finally the long-term correction of immunosuppression.

Study Design


SCID, X Linked


Gene-modified autologous stem cells


Capital Institute of Pediatrics affiliated Children's hospital


Not yet recruiting


Shenzhen Geno-Immune Medical Institute

Results (where available)

View Results


Published on BioPortfolio: 2017-07-17T09:53:50-0400

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Medical and Biotech [MESH] Definitions

Mice homozygous for the mutant autosomal recessive gene "scid" which is located on the centromeric end of chromosome 16. These mice lack mature, functional lymphocytes and are thus highly susceptible to lethal opportunistic infections if not chronically treated with antibiotics. The lack of B- and T-cell immunity resembles severe combined immunodeficiency (SCID) syndrome in human infants. SCID mice are useful as animal models since they are receptive to implantation of a human immune system producing SCID-human (SCID-hu) hematochimeric mice.

The transfer of STEM CELLS from one individual to another within the same species (TRANSPLANTATION, HOMOLOGOUS) or between species (XENOTRANSPLANTATION), or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). The source and location of the stem cells determines their potency or pluripotency to differentiate into various cell types.

Methods of implanting a CELL NUCLEUS from a donor cell into an enucleated acceptor cell. Often the nucleus of a somatic cell is transferred into a recipient OVUM or stem cell (STEM CELLS) with the nucleus removed. This technology may provide means to generate autologous diploid pluripotent cell for therapeutic cloning, and a model for studying NUCLEAR REPROGRAMMING in embryonic stem cells. Nuclear transfer was first accomplished with frog eggs (RANA PIPIENS) and reported in 1952.

Specialized stem cells that are committed to give rise to cells that have a particular function; examples are MYOBLASTS; MYELOID PROGENITOR CELLS; and skin stem cells. (Stem Cells: A Primer [Internet]. Bethesda (MD): National Institutes of Health (US); 2000 May [cited 2002 Apr 5]. Available from:

Transfer of MESENCHYMAL STEM CELLS between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS).

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