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Science Translational Medicine
|
April 30, 2010
Cystic fibrosis pigs develop lung disease and exhibit defective bacterial eradication at birth
David A Stoltz, David K Meyerholz, Alejandro A Pezzulo, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 27, 2021
Expanded phenotype of AARS1-related white matter disease
Guy Helman, Marisa I Mendes, Francesco Nicita, et al.
Human Mutation
|
December 2, 2017
Inactivation of AMMECR1 is associated with growth, bone, and heart alterations
Mariana Moysés-Oliveira, Giuliana Giannuzzi, Richard J Fish, et al.
The Journal of Clinical Investigation
|
May 17, 2013
Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs
David A Stoltz, Tatiana Rokhlina, Sarah E Ernst, et al.
American Journal of Human Genetics
|
April 16, 2019
Bi-allelic CSF1R Mutations Cause Skeletal Dysplasia of Dysosteosclerosis-Pyle Disease Spectrum and Degenerative Encephalopathy with Brain Malformation
Long Guo, Débora Romeo Bertola, Asako Takanohashi, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 12, 2025
<i>BLOC1S1</i> variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos Dominguez Gonzalez, Chad D Williamson, et al.
American Journal of Human Genetics
|
March 26, 2026
BLOC1S1 variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos A Dominguez Gonzalez, Chad D Williamson, et al.
JAMA Pediatrics
|
September 27, 2021
Effect of Whole-Genome Sequencing on the Clinical Management of Acutely Ill Infants With Suspected Genetic Disease: A Randomized Clinical Trial
, Ian D Krantz, Livija Medne, et al.
Med (New York, N.Y.)
|
February 12, 2021
Fatal perinatal mitochondrial cardiac failure caused by recurrent <i>de novo</i> duplications in the <i>ATAD3</i> locus
Ann E Frazier, Alison G Compton, Yoshihito Kishita, et al.
JAMA
|
October 24, 2024
Expanded Newborn Screening Using Genome Sequencing for Early Actionable Conditions
Alban Ziegler, Carrie Koval-Burt, Denise M Kay, et al.
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of 32
Search research articles
Search
Showing results (301-310 of 318) with videos related to
Sort By:
Page
of 32
Science Translational Medicine
|
April 30, 2010
Cystic fibrosis pigs develop lung disease and exhibit defective bacterial eradication at birth
David A Stoltz, David K Meyerholz, Alejandro A Pezzulo, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 27, 2021
Expanded phenotype of AARS1-related white matter disease
Guy Helman, Marisa I Mendes, Francesco Nicita, et al.
Human Mutation
|
December 2, 2017
Inactivation of AMMECR1 is associated with growth, bone, and heart alterations
Mariana Moysés-Oliveira, Giuliana Giannuzzi, Richard J Fish, et al.
The Journal of Clinical Investigation
|
May 17, 2013
Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs
David A Stoltz, Tatiana Rokhlina, Sarah E Ernst, et al.
American Journal of Human Genetics
|
April 16, 2019
Bi-allelic CSF1R Mutations Cause Skeletal Dysplasia of Dysosteosclerosis-Pyle Disease Spectrum and Degenerative Encephalopathy with Brain Malformation
Long Guo, Débora Romeo Bertola, Asako Takanohashi, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 12, 2025
<i>BLOC1S1</i> variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos Dominguez Gonzalez, Chad D Williamson, et al.
American Journal of Human Genetics
|
March 26, 2026
BLOC1S1 variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos A Dominguez Gonzalez, Chad D Williamson, et al.
JAMA Pediatrics
|
September 27, 2021
Effect of Whole-Genome Sequencing on the Clinical Management of Acutely Ill Infants With Suspected Genetic Disease: A Randomized Clinical Trial
, Ian D Krantz, Livija Medne, et al.
Med (New York, N.Y.)
|
February 12, 2021
Fatal perinatal mitochondrial cardiac failure caused by recurrent <i>de novo</i> duplications in the <i>ATAD3</i> locus
Ann E Frazier, Alison G Compton, Yoshihito Kishita, et al.
JAMA
|
October 24, 2024
Expanded Newborn Screening Using Genome Sequencing for Early Actionable Conditions
Alban Ziegler, Carrie Koval-Burt, Denise M Kay, et al.
Page
of 32