共 50 条
[21]
Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
[J].
Zhang, Shengwen
;
Bastille, Amy
;
Gordo, Susana
;
Ramesh, Nikhil
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Vora, Jenisha
;
McCarthy, Elizabeth
;
Zhang, Xiaohan
;
Frank, Dylan
;
Ko, Chih-Wei
;
Wu, Carmen
;
Walsh, Noel
;
Amarwani, Shreya
;
Liao, Jing
;
Xiong, Qiang
;
Drouin, Lauren
;
Hebben, Matthias
;
Chiang, Kyle
;
Chau, B. Nelson
.
PLOS ONE,
2022, 17 (09)

Zhang, Shengwen
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Bastille, Amy
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Gordo, Susana
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Ramesh, Nikhil
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Vora, Jenisha
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

McCarthy, Elizabeth
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Zhang, Xiaohan
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Frank, Dylan
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Ko, Chih-Wei
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Wu, Carmen
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Walsh, Noel
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Amarwani, Shreya
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Liao, Jing
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Xiong, Qiang
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Drouin, Lauren
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Hebben, Matthias
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Chiang, Kyle
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA

Chau, B. Nelson
论文数: 0 引用数: 0
h-index: 0
机构:
LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA
[22]
Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
[J].
Koji M. Nishiguchi
;
Kosuke Fujita
;
Fuyuki Miya
;
Shota Katayama
;
Toru Nakazawa
.
Nature Communications,
11

Koji M. Nishiguchi
论文数: 0 引用数: 0
h-index: 0
机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine

Kosuke Fujita
论文数: 0 引用数: 0
h-index: 0
机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine

Fuyuki Miya
论文数: 0 引用数: 0
h-index: 0
机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine

Shota Katayama
论文数: 0 引用数: 0
h-index: 0
机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine

Toru Nakazawa
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h-index: 0
机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine
[23]
Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization
[J].
Chung, Sook Hyun
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Mollhoff, Iris Natalie
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Nguyen, Uyen
;
Nguyen, Amy
;
Stucka, Natalie
;
Tieu, Eric
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Manna, Suman
;
Meleppat, Ratheesh Kumar
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Zhang, Pengfei
;
Nguyen, Emerald Lovece
;
Fong, Jared
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Zawadzki, Robert
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Yiu, Glenn
.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT,
2020, 17
:409-417

Chung, Sook Hyun
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Mollhoff, Iris Natalie
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Nguyen, Uyen
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Nguyen, Amy
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Stucka, Natalie
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Tieu, Eric
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Manna, Suman
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Meleppat, Ratheesh Kumar
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Zhang, Pengfei
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Nguyen, Emerald Lovece
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Fong, Jared
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Zawadzki, Robert
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h-index: 0
机构:
Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA

Yiu, Glenn
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h-index: 0
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Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA
[24]
SUSTAINED HIGH LEVEL EXPRESSION OF FVII FOLLOWING AAV-MEDIATED GENE DELIVERY IN VIVO
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D'Amico, A.
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Hobo, B.
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HAEMOPHILIA,
2023, 29
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D'Amico, A.
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h-index: 0
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Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Blits, B.
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h-index: 0
机构:
SanaGen BV, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Van Den Herik, J.
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机构:
Netherlands Inst Neurosci, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

De Winter, F.
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机构:
Netherlands Inst Neurosci, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Kaijen, P.
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h-index: 0
机构:
Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Hobo, B.
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h-index: 0
机构:
Netherlands Inst Neurosci, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Verhaagen, J.
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h-index: 0
机构:
Netherlands Inst Neurosci, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Mosmuller, D.
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机构:
SanaGen BV, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands

Voorberg, J.
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Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands Sanquin Res, Deptartment Mol Hematol, Amsterdam, Netherlands
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Emerging Issues in AAV-Mediated In Vivo Gene Therapy
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Colella, Pasqualina
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MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT,
2018, 8
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Colella, Pasqualina
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h-index: 0
机构:
Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France

Ronzitti, Giuseppe
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h-index: 0
机构:
Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France

Mingozzi, Federico
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h-index: 0
机构:
Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France
Univ Pierre & Marie Curie Paris 6, F-75651 Paris, France
INSERM U974, F-75651 Paris, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France
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AAV-MEDIATED, IN VIVO GENE DELIVERY TO THE ROTATOR CUFF
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Sherwin, R. E.
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2023, 46
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Sherwin, R. E.
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h-index: 0
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Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA
Mayo Clin, Musculoskeletal Gene Therapy Res Lab, 200 First St SW, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Mcglinch, E. B.
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h-index: 0
机构:
Mayo Clin, Musculoskeletal Gene Therapy Res Lab, 200 First St SW, Rochester, MN 55905 USA
Mayo Clin, Grad Sch Biomed Sci, Rochester, MN 55905 USA
Mayo Clin, Virol & Gene Therapy Grad Program, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Barry, M. A.
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h-index: 0
机构:
Mayo Clin, Dept Med, Rochester, MN 55905 USA
Mayo Clin, Dept Mol Med, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Padilla, C. M. Lopez De
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h-index: 0
机构:
Mayo Clin, Musculoskeletal Gene Therapy Res Lab, 200 First St SW, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Montonye, D. R.
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h-index: 0
机构:
Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Evans, C. H.
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h-index: 0
机构:
Mayo Clin, Musculoskeletal Gene Therapy Res Lab, 200 First St SW, Rochester, MN 55905 USA Mayo Clin, Dept Comparat Med, Rochester, MN 55905 USA

Atasoy-Zeybek, A.
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h-index: 0
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Standard screening methods underreport AAV-mediated transduction and gene editing
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Brida, Kasey L.
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NATURE COMMUNICATIONS,
2019, 10 (1)

Lang, Jonathan F.
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h-index: 0
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Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Toulmin, Sushila A.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Brida, Kasey L.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Eisenlohr, Laurence C.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Davidson, Beverly L.
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h-index: 0
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Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
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Beverly L. Davidson
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Nature Communications,
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Jonathan F. Lang
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h-index: 0
机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of Medicine

Sushila A. Toulmin
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h-index: 0
机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of Medicine

Kasey L. Brida
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h-index: 0
机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of Medicine

Laurence C. Eisenlohr
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h-index: 0
机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of Medicine

Beverly L. Davidson
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Zhang, S.
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McCarthy, E.
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Zhang, S.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Bastille, A.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Ramesh, N.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

McCarthy, E.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Vora, J.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Ko, C.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Wu, C.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Walsh, N.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Frank, D.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Wang, C. C.
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Wang, S.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Wang, J.
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Freland, L.
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Liao, J.
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Gordo, S.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Xiong, Q.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Drouin, L.
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h-index: 0
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Preston, G.
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h-index: 0
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Hebben, M.
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h-index: 0
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Chiang, K.
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LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

Chau, N.
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h-index: 0
机构:
LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA

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2025, 16 (01)

Ferriero, Rosa
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h-index: 0
机构:
Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Bruno, Gemma
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h-index: 0
机构:
Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Padula, Agnese
论文数: 0 引用数: 0
h-index: 0
机构:
Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Pisano, Simone
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h-index: 0
机构:
Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy
European Sch Mol Med SEMM, Milan, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Boffa, Iolanda
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h-index: 0
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Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Gargaro, Marco
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h-index: 0
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Univ Perugia, Dept Med & Surg, Perugia, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy

Imperatore, Teresa
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h-index: 0
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Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy
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Battipaglia, Maria
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Vivenzio, Silvia
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Perna, Claudia
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Nusco, Edoardo
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Ferrante, Luigi
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Westhaus, Adrian
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Knight, Maddison
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Manni, Giorgia
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Campione, Severo
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Di Napoli, Evaristo
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Polishchuk, Elena
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Polishchuk, Roman
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Paciello, Orlando
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Brunetti-Pierri, Nicola
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Lisowski, Leszek
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Piccolo, Pasquale
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