PEG‐ADA: An alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency

Human Mutation

Volumn 5, Issue 2, 1995, Pages 107-112

  Hershfield, Michael S ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Enzyme replacement therapy; Immunodeficiency; Orphan drug; Polyethylene glycol

Indexed keywords

ADENOSINE DEAMINASE; MACROGOL; ORPHAN DRUG;

BONE MARROW TRANSPLANTATION; COMBINED IMMUNODEFICIENCY; COST BENEFIT ANALYSIS; ENZYME DEFICIENCY; GENE THERAPY; HLA SYSTEM; HUMAN; IMMUNE FUNCTION TEST; INFECTION RISK; OPPORTUNISTIC INFECTION; PRIORITY JOURNAL; REVIEW; RISK ASSESSMENT; SOMATIC CELL GENETICS;

ADENOSINE DEAMINASE; ADOLESCENT; ADULT; BONE MARROW TRANSPLANTATION; CHILD; COMPARATIVE STUDY; GENE THERAPY; HAPLOTYPES; HUMAN; INFANT; SEVERE COMBINED IMMUNODEFICIENCY;

ADA;

EID: 0028907019     PISSN: 10597794     EISSN: 10981004     Source Type: Journal    
DOI: 10.1002/humu.1380050202     Document Type: Review

References (22)

1. Correct splicing despite a G>A mutation at the invariant first nucleotide ot a 5′ splice site: A possible basis for disparate clinical phenotypes in siblings with adenosine deaminase (ADA) deficiency
2. Development of gene therapy tor immunodeficiency: Adenosine deaminase deficiency
3. Comparison of red cell transfusion and polyethylene glycol‐modified adenosine deaminase therapy in an adenosine deaminase‐deficient child
4. IgG antibody response to polyethylene glycol‐modified adenosine deaminase (PEG‐ADA) in patients with adenosine deaminase deficiency
5. Suppression of an antibody to adenosine deaminase (ADA) in an ADA deficient severe combined immunodeficiency patient receiving polyethylene glycol modified adenosine deaminase (PEG‐ADA)
6. Alteration of the circulating life and antigenic properties of bovine adenosine deaminase in mice by attachment of polyethylene glycol
7. Immune reconstitution in severe combined immunodeficiency disease after lectin‐treated, T‐cell‐depleted haplocompatible bone marrow transplantation
8. European experience of bone marrow transplantation for severe combined immunodeficiency
9. Traitement du deficit en adenosine desaminase par l'adenosine desaminase couplee au polyethylene glycol (PEG‐ADA)
10. Treatment of adenosine deaminase deficiency with polyethylene glycol‐modified adenosine deaminase
11. Enzyme replacement therapy with PEG‐ADA in adenosine deaminase deficiency: Overview and case reports of three patients, including two now receiving gene therapy
12. Immunodeficiency diseases caused by adenosine deaminase deficiency and purine nucleoside phosphorylase deficiency
13. Overview of biochemical abnormalities and molecular genetics of adenosine deaminase deficiency
14. Adenosine deaminase deficiency with late onset of recurrent infections: Response to treatment with polyethylene glycol‐modified adenosine deaminase (PEG‐ADA)
15. Insulin‐dependent diabetes mellitus and severe atopic dermatitis in a child with adenosine deaminase deficiency
16. Antibody responses to bacteriophage ØX174 in patients with adenosine deaminase deficiency
17. The use of HLA‐non‐identical T‐cell‐depleted marrow for transplants for correction of severe combined immunodeficiency disease
18. Novel splicing, missense, and deletion mutations in 7 adenosine deaminase deficient patients with late/delayed onset of combined immunodeficiency disease: Contribution of genotype to phenotype
19. Severe combined immunodeficiency: A retrospective single center study of clinical presentation and outcome in 117 patients
20. Heterogeneity of phenotype in two siblings with adenosine deaminase deficiency
21. Failure to thrive, cough and oral candidiasis in a three month old male
22. T lymphocyte ontogeny in adenosine deaminase deficient severe cobined immune deficiency following treatment with polyethylene glycol modified adenosine deaminase