Gene therapy for SCID

The current treatment by allogeneic hematopoietic stem cell transplantation (HSCT) is effective but carries the risk of graft-versus-host disease. This ...

Gene therapy for severe combined immunodeficiencies and beyond

SCIDs can be successfully treated with allogeneic HSCT, which provides long-term correction of the T cell deficiency. In the early 1990s, ...

Gene Therapy Cures SCID | St. Jude Research

Gene therapy developed at St. Jude is driving a landmark cure for patients with X-linked severe combined immunodeficiency (SCID-X1). Sometimes called "bubble ...

Gene Therapy for Severe Combined Immunodeficiency (SCID)

Gene therapies for SCID have been explored. It involves the isolation and molecular correction of mutations in the patients own haematological stem cells.

Severe Combined Immunodeficiency (SCID)

Even though SCID is a genetic condition, having one child with SCID does not necessarily mean other family members, including siblings, are going to develop the ...

Severe Combined Immunodeficiency (SCID) and Its New Treatment ...

The various treatment modalities include hematopoietic stem cell transplantation, gene therapy, enzyme replacement therapy and chemotherapy.

Efficacy of Gene Therapy for X-Linked Severe Combined ...

After nearly 10 years of follow-up, gene therapy was shown to have corrected the immunodeficiency associated with SCID-X1.

Gene therapy for ADA-SCID shows promise

48 out of 50 infants and children with ADA-SCID treated with gene therapy experienced a restored immune system within a period of two to three years.

Severe Combined Immunodeficiency (SCID) Treatment ...

An alternative approach that has been increasingly applied to SCID in recent decades is gene therapy: the transfer of normal genes into ...

Gene therapy offers a potential cure to children born without an ...

The standard treatment for ADA-SCID involves once or twice weekly injections of the ADA enzyme until a matched bone marrow donor – usually a ...

Treating the bubble babies: gene therapy in use - Your Genome

Severe combined immunodeficiency (SCID) is a genetic disorder characterised by a reduced number of immune cells. Some children with the condition have been ...

3D genome architecture influences SCID-X1 gene therapy success

Several years after treatment, the St. Jude lentiviral gene therapy for SCID-X1 appears both effective and safe, unlike earlier retroviral ...

SCID gene therapy trial publishes results

A new 'robust' gene therapy for severe combined immunodeficiency (SCID), a condition which leaves children without an immune system, has been ...

Gene therapy | Immune Deficiency Foundation

Gene therapy is still in clinical trials for several types of primary immunodeficiency (PI), including severe combined immunodeficiency (SCID), ...

Gene therapy for X-linked severe combined immunodeficiency

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Immune Reconstitution After Gene Therapy Approaches in Patients ...

Gene therapy used for SCID-X1 clinical trials so far has been based on the use of retroviral vectors to transfer a corrective copy of the defective gene to ...

Early success for gene therapy against severe combined ... - YouTube

Clinical trial results suggest gene therapy might offer a promising option for some patients with X-linked severe combined immunodeficiency ...

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in ...

The use of busulfan conditioning in allogeneic hematopoietic stem-cell transplantation in infants with all genetic forms of SCID has been ...

Gene Therapy for XSCID: The First Success of Gene Therapy - Nature

A recent report by Cavazzana-Calvo et al. (4) validates the concept of SCID as a favorable model disease for early gene therapy studies and ...

Integrome signatures of lentiviral gene therapy for SCID-X1 patients

Our findings suggest that LV integrome signatures, shaped by common features such as genome organization, may affect the efficacy of LV-based cellular ...