Gene therapy targeting hematopoietic cells: better not leave it to chance.

Abstract:

:Gene therapy targeting hematopoietic cells has arrived at a new stage of potency. While the potential for curing inherited disorders of the immune system has been demonstrated in clinical trials, we were also confronted with the first serious adverse events related to random insertion of foreign DNA into cellular chromosomes. As it is likely that the manifestation of severe side effects results from a multifactorial process, it will be of crucial importance to define the significance of the individual risk factors involved. The future of the field will depend on our ability to define risk classifications of clinical approaches, to continuously improve transgene technologies, and to introduce new concepts for targeted selection of transgenic cells. Interestingly, correction of genetic disorders by homologous gene repair in defined stem cell clones is on the horizon, but far from being available for clinical use.

journal_name

Acta Haematol

journal_title

Acta haematologica

authors

Baum C,von Kalle C

doi

10.1159/000072459

subject

Has Abstract

pub_date

2003-01-01 00:00:00

pages

107-9

issue

2-3

eissn

0001-5792

issn

1421-9662

pii

72459

journal_volume

110

pub_type

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