1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological Reconstitution

Otsu, Makoto; Nakajima, Satoru; Kida, Masataka; Maeyama, Yoshihiro; Toita, Nariaki; Hatano, Norikazu; Kawamura, Nobuaki; Kobayashi, Riko; Tatsuzawa, Osamu; Onodera, Masafumi; Candotti, Fabio; Bali, Pawan; Hershfield, Michael S.; Sakiyama, Yukio; Ariga, Tadashi

doi:10.1016/j.ymthe.2006.08.1190

Cited by 4 publications

(3 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In this case, PEG-ADA was discontinued to enhance selective advantage, but no cytoreductive preparative regimen was given. The investigators reported a delayed immune reconstitution and effective detoxification of purine metabolites, although no information on engraftment of HSC was available [38].…”

Section: Other Gene Therapy Trials For Ada-scidmentioning

confidence: 99%

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

et al. 2009

View full text Add to dashboard Cite

Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors. The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal purine metabolism and immune functions. Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system.

show abstract

Section: Other Gene Therapy Trials For Ada-scidmentioning

confidence: 99%

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

et al. 2009

View full text Add to dashboard Cite

show abstract

“…Although some degree of immunological reconstitution was obtained in two patients, a longer follow-up to determine HSC engraftment and the efficacy of systemic detoxification is required [26].…”

Section: Long-term Outcome Of Gene Therapy With Nonmyeloablative Condmentioning

confidence: 99%

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency

Ferrua

Brigida

Aiuti

2010

Current Opinion in Allergy &Amp; Clinical Immunology

View full text Add to dashboard Cite

show abstract

“…Cappelli & Aiuti 2010, Ferrua et al 2010a). 15 patients were treated with retrovirally transduced autologous HSC in HSR-TIGET, Italy, Aiuti et al 2002, 10 in CHLA-NIH, USA(Engel et al 2007, Sokolic et al 2008, 5 in GOSH, UK(Gaspar et al 2009, Gaspar et al 2006, and 2 in Hokkaido, Japan(Otsu et al 2006). The patients from the Italy trial displayed progressive immune reconstitution and long-term multilineage engraftment and were able to discontinue ERT treatment in 13 of the 15 cases.…”

mentioning

confidence: 99%

Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases and Its Implications for Future Gene Therapy Trials in Turkey

Aerts‐Kaya

Esken

Erol

2019

Trakya University Journal of Natural Sciences

View full text Add to dashboard Cite

Stem cell therapy offers a great advantage for the development of new treatments in the field of regenerative and restorative medicine. However, the use of stem cell therapies and their clinical indications can even be further improved using genetic modification of the cells. Due to the high level of consanguineous marriages in Turkey, the country suffers from an increased frequency of inborn genetically inherited diseases. Treatment of these diseases is difficult, since 1) diagnosis is often delayed in rural areas, 2) distance to specialized centers may be considerable, 3) treatment may require frequent hospital visits and 4) treatment procedures are often both invasive and expensive. Here, we discuss the current status of gene therapy of hematopoietic stem cells (HSCs) for rare, inherited monogenic diseases and the advantages to use these cells as an alternative treatment option for patients in Turkey. We discuss results of clinical trials using retroviral and lentiviral gene therapy for the treatment of immune deficiencies, hemoglobinopathies and several enzyme deficiencies, new developments in the field of the HSC gene therapy to improve safety and efficacy and recommendations for the future.

show abstract

1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological Reconstitution

Cited by 4 publications

References 0 publications

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency

Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases and Its Implications for Future Gene Therapy Trials in Turkey

Contact Info

Product

Resources

About