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C Ahrens

Showing results (131-140 of 169) with videos related to

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Molecular Therapy : the Journal of the American Society of Gene Therapy|December 30, 2025
Response to the FDA's proposed pathway for individualized genetic therapiesTimothy W Yu, Julia Vitarello, Kiran Musunuru, et al.
Biorxiv : the Preprint Server for Biology|May 7, 2024
Improved specificity and efficacy of base-editing therapies with hybrid guide RNAsMadelynn N Whittaker, Dominique L Brooks, Aidan Quigley, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|September 19, 2019
Clinical utility of exome sequencing in infantile heart failureAlyssa Ritter, Emma Bedoukian, Justin H Berger, et al.
American Journal of Respiratory and Critical Care Medicine|May 10, 2008
Intersession variability in single-breath diffusing capacity in diabetics without overt lung diseaseMichael B Drummond, Pamela F Schwartz, William T Duggan, et al.
The Journal of Pediatrics|November 3, 2023
Diagnostic Yield of Exome Sequencing in Pediatric CardiomyopathyJulia Keisling, Emma Bedoukian, Danielle S Burstein, et al.
Pediatric Pulmonology|January 22, 2002
Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosisRichard C Ahrens, Thomas A Standaert, Janice Launspach, et al.
International Journal of Neonatal Screening|April 25, 2022
Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in PennsylvaniaJessica R C Priestley, Laura A Adang, Sarah Drewes Williams, et al.
Neurology. Genetics|February 10, 2017
Precision therapy for a new disorder of AMPA receptor recycling due to mutations in <i>ATAD1</i>Rebecca C Ahrens-Nicklas, George K E Umanah, Neal Sondheimer, et al.
Pediatric Pulmonology|January 22, 2002
A phase I randomized, multicenter trial of CPX in adult subjects with mild cystic fibrosisNael A McCarty, Thomas A Standaert, Mary Teresi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 22, 2024
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiencyVi Pham, Lucas Tricoli, Xinying Hong, et al.
Pageof 17

Showing results (131-140 of 169) with videos related to

Sort By:
Pageof 17
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 30, 2025
Response to the FDA's proposed pathway for individualized genetic therapiesTimothy W Yu, Julia Vitarello, Kiran Musunuru, et al.
Biorxiv : the Preprint Server for Biology|May 7, 2024
Improved specificity and efficacy of base-editing therapies with hybrid guide RNAsMadelynn N Whittaker, Dominique L Brooks, Aidan Quigley, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|September 19, 2019
Clinical utility of exome sequencing in infantile heart failureAlyssa Ritter, Emma Bedoukian, Justin H Berger, et al.
American Journal of Respiratory and Critical Care Medicine|May 10, 2008
Intersession variability in single-breath diffusing capacity in diabetics without overt lung diseaseMichael B Drummond, Pamela F Schwartz, William T Duggan, et al.
The Journal of Pediatrics|November 3, 2023
Diagnostic Yield of Exome Sequencing in Pediatric CardiomyopathyJulia Keisling, Emma Bedoukian, Danielle S Burstein, et al.
Pediatric Pulmonology|January 22, 2002
Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosisRichard C Ahrens, Thomas A Standaert, Janice Launspach, et al.
International Journal of Neonatal Screening|April 25, 2022
Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in PennsylvaniaJessica R C Priestley, Laura A Adang, Sarah Drewes Williams, et al.
Neurology. Genetics|February 10, 2017
Precision therapy for a new disorder of AMPA receptor recycling due to mutations in <i>ATAD1</i>Rebecca C Ahrens-Nicklas, George K E Umanah, Neal Sondheimer, et al.
Pediatric Pulmonology|January 22, 2002
A phase I randomized, multicenter trial of CPX in adult subjects with mild cystic fibrosisNael A McCarty, Thomas A Standaert, Mary Teresi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 22, 2024
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiencyVi Pham, Lucas Tricoli, Xinying Hong, et al.
Pageof 17