In utero transplantation of monocytic cells in cats with alpha-mannosidosis.

ABSTRACT:

Background

Lysosomal storage diseases are devastating illnesses, in large part because of their neurologic consequences. Because significant morbidity occurs prenatally, in utero (IU) therapy is an attractive therapeutic approach.

Methods

We studied the feasibility and efficacy of IU injections of monocytic cells (derived from normal marrow) in feline alpha-mannosidosis. Heterozygous cats were interbred to produce affected (homozygous) and control (heterozygous and wild-type) offspring. Thirty-seven pregnancies were studied in which fetuses were transplanted intraperitoneally (1x10 cells/kg recipient) at gestational days 27 to 33 and then each week for 2 weeks (term=63 days). After birth, affected kittens were evaluated clinically and pathologically, tissue alpha-mannosidase

SUBMITTER: Abkowitz JL 

PROVIDER: S-EPMC2742773 | biostudies-literature | 2009 Aug

REPOSITORIES: biostudies-literature