Long-Term Safety and Urea Cycle Function Following a Phase 1/2 Trial of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

被引：0

作者：

Harding, Cary O. ^{[1
]}

Geberhiwot, Tarekegn ^{[2
]}

Couce, Maria Luz ^{[3
]}

Guffon, Nathalie ^{[4
]}

Tan, Wen-Hann ^{[5
]}

Hualde, Leticia Ceberio ^{[6
]}

Khan, Aneal ^{[7
]}

Konczal, Laura ^{[8
]}

Thomas, Janet A. ^{[9
,10
]}

Breilyn, Margo Sheck ^{[11
]}

Zhang, Lin ^{[12
]}

Merritt, J. Lawrence ^{[12
]}

Crombez, Eric ^{[12
]}

机构：

[1] Oregon Hlth & Sci Univ, Portland, OR USA

[2] Univ Birmingham, Birmingham, W Midlands, England

[3] Univ Santiago Compostela, Santiago De Compostela, Spain

[4] HCL, Reference Ctr Inherited Metab Dis, Lyon, France

[5] Boston Childrens Hosp, Boston, MA USA

[6] Cruces Univ Hosp, Biocruces Bizkaia Hlth Res Inst, Baracaldo, Spain

[7] MAGic Clin Ltd, Calgary, AB, Canada

[8] Univ Hosp Cleveland Med Ctr, Cleveland, OH USA

[9] Univ CO, Sch Med, Aurora, CO USA

[10] Childrens Hosp Colorado, Aurora, CO USA

[11] Icahn Sch Med Mt Sinai, New York, NY USA

[12] Ultragenyx Pharmaceut Inc, Novato, CA USA

来源：

MOLECULAR THERAPY | 2024年 / 32卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

1411

引用

页码：668 / 669

页数：2

共 22 条

[1] Long-Term Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial
Harding, Cary O.
Geberhiwot, Tarekegn
Couce, Maria Luz
Guffon, Nathalie
Hualde, Leticia Ceberio
Khan, Aneal
Konczal, Laura
Tan, Wen-Hann
Thomas, Janet
Breilyn, Margo
Patra, Kaushik
Merritt, J. Lawrence
Crombez, Eric
MOLECULAR THERAPY, 2023, 31 (04) : 69 - 69
[2] Long-term safety and efficacy of DTX301 in adults with late-onset ornithine transcarbamylase (OTC) deficiency: A Phase 1/2 trial
Thomas, Janet
Tan, Wen-Hann
Khan, Aneal
Konczal, Laura
Breilyn, Margo Sheck
Hualde, Leticia Ceberio
Couce, Marie Luz
Geberhiwot, Tarekegn
Guffon, Nathalie
Harding, Cary
Patra, Kaushik
Merritt, J. Lawrence, II
Crombez, Eric
MOLECULAR GENETICS AND METABOLISM, 2024, 141 (04)
[3] Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial
Harding, Cary O.
Geberhiwot, Tarekegn
Couce, Maria Luz
Tan, Wen-Hann
Khan, Aneal
Hualde, Leticia Ceberio
Diaz, George A.
Konczal, Laura
Thomas, Janet
Guffon, Nathalie
Lee, Connie
Puga, Ana
Crombez, Eric
MOLECULAR THERAPY, 2022, 30 (04) : 219 - 219
[4] Final safety and efficacy of a phase 1/2 trial of DTX301 in adults with late-onset ornithine transcarbamylase deficiency (OTCD)
Geberhiwot, T.
Harding, C. O.
Couce, M. L.
Tan, W-H
Khan, A.
Hualde, L. C.
Diaz, G. A.
Konczal, L.
Thomas, J.
Guffon, N.
Lee, C.
Puga, A. C.
Crombez, E.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A9 - A9
[5] AAV8 gene therapy as a potential treatment for adults with late-onset ornithine transcarbamylase (OTC) deficiency: updated results from a phase 1/2 clinical trial
Geberhiwot, T.
Couce, M. L.
Hualde, L. C.
Harding, C. O.
Khan, A.
Diaz, G. A.
Tan, W.
Konczal, L.
Thomas, J.
Guffon, N.
Lee, C.
Puga, A. C.
Crombez, E.
HUMAN GENE THERAPY, 2021, 32 (19-20) : A9 - A9
[6] AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: Updated Results from a Phase 1/2 Clinical Trial
Harding, Cary O.
Luz Couce, Maria
Geberhiwot, Tarekegn
Tan, Wen-Hann
Khan, Aneal
Aldamiz-Echevarria, Luis
Diaz, George A.
Lee, Connie
Puga, Ana Cristina
Crombez, Eric
MOLECULAR THERAPY, 2021, 29 (04) : 17 - 17
[7] Effects of arginine treatment on nutrition, growth and urea cycle function in seven Japanese boys with late-onset ornithine transcarbamylase deficiency
Hironori Nagasaka
Tohru Yorifuji
Kei Murayama
Mitsuru Kubota
Keiji Kurokawa
Tomoko Murakami
Masaki Kanazawa
Tomozumi Takatani
Atsushi Ogawa
Emi Ogawa
Shigenori Yamamoto
Masanori Adachi
Kunihiko Kobayashi
Masaki Takayanagi
European Journal of Pediatrics, 2006, 165 : 618 - 624
[8] Effects of arginine treatment on nutrition, growth and urea cycle function in seven Japanese boys with late-onset ornithine transcarbamylase deficiency
Nagasaka, Hironori
Yorifuji, Tohru
Murayama, Kei
Kubota, Mitsuru
Kurokawa, Keiji
Murakami, Tomoko
Kanazawa, Masaki
Takatani, Tomozumi
Ogawa, Atsushi
Ogawa, Emi
Yamamoto, Shigenori
Adachi, Masanori
Kobayashi, Kunihiko
Takayanagi, Masaki
EUROPEAN JOURNAL OF PEDIATRICS, 2006, 165 (09) : 618 - 624
[9] A phase 1/2 clinical trial for AAV8-mediated liver-directed gene therapy in adults with late-onset OTC deficiency
Geberhiwot, T.
Aldamiz-Echevarria, L.
Couce, M. L.
Harding, C. O.
Khan, A.
Tan, W. H.
Lin, J.
Crombez, E.
Diaz, G. A.
HUMAN GENE THERAPY, 2018, 29 (12) : A59 - A59
[10] AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset OTC Deficiency: Results from a Phase 1/2 Clinical Trial
Tan, Wen-Hann
Geberhiwot, Tarekegn
Couce, Maria Luz
Aldamiz-Echevarria, Luis
Diaz, George A.
Harding, Cary O.
Khan, Aneal
Lee, Connie
Puga, Ana Cristina
Crombez, Eric
MOLECULAR THERAPY, 2020, 28 (04) : 222 - 222

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