Due to their underlying genetic complexity, chromosomal disorders such as Down syndrome (DS), which is caused by trisomy 21, have long been understudied and continue to lack effective treatments. With over 200 genes on the extra chromosome, even the specific cell pathologies and pathways impacted in DS are not known, and it has not been considered a viable target for the burgeoning field of gene therapy. Recently, our lab demonstrated that the natural mechanism of dosage compensation can be harnessed to silence the trisomic chromosome in pluripotent cells. Using an inducible XIST transgene allows us to study the effects of trisomy in a tightly controlled system by comparing the same cells with either two or three active copies of chromosome...
<div><p>Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter exp...
IntroductionDown syndrome, caused by trisomy 21, is a complex developmental disorder associated with...
Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter expression ...
Down syndrome is the leading genetic cause of intellectual disabilities, occurring in 1 out of 700 l...
The ability of XIST to dosage compensate a trisomic autosome presents unique experimental opportunit...
Down syndrome (DS) is a common disorder with enormous medical and social costs, caused by trisomy fo...
Down syndrome (DS) is a common disorder with enormous medical and social costs, caused by trisomy fo...
Maintenance of gene dosage is important for proper cellular function and development, as evidenced b...
Down syndrome is the most common genetic cause of intellectual disability worldwide and affects more...
SummaryHuman trisomies can alter cellular phenotypes and produce congenital abnormalities such as Do...
We previously demonstrated that an integrated XIST transgene can broadly repress one chromosome 21 i...
Down syndrome (DS) is the most frequent cause of human congenital mental retardation. Cognitive defi...
Down syndrome (trisomy 21) is the most common viable chromosomal disorder with intellectual impairme...
Down Syndrome (DS), or Trisomy 21 Syndrome, is one of the most common genetic diseases. It is a chro...
BACKGROUND: Down syndrome (DS) individuals suffer mental retardation with further cognitive decline ...
<div><p>Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter exp...
IntroductionDown syndrome, caused by trisomy 21, is a complex developmental disorder associated with...
Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter expression ...
Down syndrome is the leading genetic cause of intellectual disabilities, occurring in 1 out of 700 l...
The ability of XIST to dosage compensate a trisomic autosome presents unique experimental opportunit...
Down syndrome (DS) is a common disorder with enormous medical and social costs, caused by trisomy fo...
Down syndrome (DS) is a common disorder with enormous medical and social costs, caused by trisomy fo...
Maintenance of gene dosage is important for proper cellular function and development, as evidenced b...
Down syndrome is the most common genetic cause of intellectual disability worldwide and affects more...
SummaryHuman trisomies can alter cellular phenotypes and produce congenital abnormalities such as Do...
We previously demonstrated that an integrated XIST transgene can broadly repress one chromosome 21 i...
Down syndrome (DS) is the most frequent cause of human congenital mental retardation. Cognitive defi...
Down syndrome (trisomy 21) is the most common viable chromosomal disorder with intellectual impairme...
Down Syndrome (DS), or Trisomy 21 Syndrome, is one of the most common genetic diseases. It is a chro...
BACKGROUND: Down syndrome (DS) individuals suffer mental retardation with further cognitive decline ...
<div><p>Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter exp...
IntroductionDown syndrome, caused by trisomy 21, is a complex developmental disorder associated with...
Trisomy of chromosome 21, the genetic cause of Down syndrome, has the potential to alter expression ...