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AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.234-245

2020

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Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

PloS one, 2021-01, Vol.16 (1), p.e0245831-e0245831

2021

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High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU)

Molecular genetics and metabolism, 2016-01, Vol.117 (1), p.5-11

2016

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Pharmacologic inhibition of L-tyrosine degradation ameliorates cerebral dopamine deficiency in murine phenylketonuria (PKU)

Journal of inherited metabolic disease, 2014-09, Vol.37 (5), p.735-743

2014

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Hepatocytes from wild-type or heterozygous donors are equally effective in achieving successful therapeutic liver repopulation in murine phenylketonuria (PKU)

Molecular genetics and metabolism, 2011-11, Vol.104 (3), p.235-240

2011

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Bone morphogenetic proteins: An update on basic biology and clinical relevance

Journal of orthopaedic research, 1999-03, Vol.17 (2), p.269-278

1999

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Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes

Science translational medicine, 2021-06, Vol.13 (597)

2021

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Modeling the cognitive effects of diet discontinuation in adults with phenylketonuria (PKU) using pegvaliase therapy in PAH-deficient mice

Molecular genetics and metabolism, 2022-05, Vol.136 (1), p.46-64

2022

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A novel Pah-exon1 deleted murine model of phenylalanine hydroxylase (PAH) deficiency

Molecular genetics and metabolism, 2020-11, Vol.131 (3), p.306-315

2020

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Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice

Molecular genetics and metabolism, 2018-01, Vol.123 (1), p.6-20

2018

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Biochemical and behavioural profile of NTBC treated Tyrosinemie type 1 mice

Molecular genetics and metabolism, 2022-09, Vol.137 (1-2), p.9-17

2022

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Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease

Nature (London), 1997-03, Vol.386 (6623), p.395-399

1997

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VEGF Gene Therapy Augments Localized Angiogenesis and Promotes Anastomotic Wound Healing: A Pilot Study in a Clinically Relevant Animal Model

Journal of gastrointestinal surgery, 2008-10, Vol.12 (10), p.1762-1772

2008

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Managing chronic pain with encapsulated cell implants releasing catecholamines and endogenous opiods

Frontiers in bioscience, 2005-01, Vol.10 (1-3), p.367

2005

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Tetrahydrobiopterin treatment reduces brain L-Phe but only partially improves serotonin in hyperphenylalaninemic ENU1/2 mice

Journal of inherited metabolic disease, 2018-07, Vol.41 (4), p.709-718

2018

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Porous polymer scaffolds surface-modified with arginine-glycine-aspartic acid enhance bone cell attachment and differentiation in vitro

Journal of biomedical materials research, 2003-03, Vol.64A (3), p.583-590

2003

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Neuroprotective effects of encapsulated CNTF-producing cells in a rodent model of Huntington's disease are dependent on the proximity of the implant to the lesioned striatum

Cell transplantation, 2004-01, Vol.13 (3), p.253-259

2004

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Reduction Mammaplasty : A Review of Managed Care Medical Policy Coverage Criteria

Plastic and reconstructive surgery (1963), 2008-04, Vol.121 (4), p.1092-1100

2008

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Sustained release emphasizing recombinant human bone morphogenetic protein-2

Advanced drug delivery reviews, 1998-05, Vol.31 (3), p.303-318

1998

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Stability of hydrogels used in cell encapsulation: An in vitro comparison of alginate and agarose

Biotechnology and bioengineering, 1996-05, Vol.50 (4), p.374-381

1996

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Manipulation of Suspended Single Cells by Microfluidics and Optical Tweezers

Cellular and molecular bioengineering, 2010-09, Vol.3 (3), p.213-228

2010

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Gene therapy approaches for modulating bone regeneration

Advanced drug delivery reviews, 2000-08, Vol.42 (1), p.121-138

2000

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Revascularization of rat fasciocutaneous flap using CROSSEAL® with VEGF protein or plasmid DNA expressing VEGF

Otolaryngology-head and neck surgery, 2008-08, Vol.139 (2), p.245-249

2008

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Injection of Chemotherapeutic Microspheres and Glioma IV: Eradicating Tumors in Rats

Cell transplantation, 2002-01, Vol.11 (1), p.47-54

2002

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Cell delivery to the central nervous system

Advanced drug delivery reviews, 2000-08, Vol.42 (1), p.81-102

2000

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AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU)

Pharmacologic inhibition of L-tyrosine degradation ameliorates cerebral dopamine deficiency in murine phenylketonuria (PKU)

Hepatocytes from wild-type or heterozygous donors are equally effective in achieving successful therapeutic liver repopulation in murine phenylketonuria (PKU)

Bone morphogenetic proteins: An update on basic biology and clinical relevance

Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes

Modeling the cognitive effects of diet discontinuation in adults with phenylketonuria (PKU) using pegvaliase therapy in PAH-deficient mice

A novel Pah-exon1 deleted murine model of phenylalanine hydroxylase (PAH) deficiency

Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice

Biochemical and behavioural profile of NTBC treated Tyrosinemie type 1 mice

Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease

VEGF Gene Therapy Augments Localized Angiogenesis and Promotes Anastomotic Wound Healing: A Pilot Study in a Clinically Relevant Animal Model

Managing chronic pain with encapsulated cell implants releasing catecholamines and endogenous opiods

Tetrahydrobiopterin treatment reduces brain L-Phe but only partially improves serotonin in hyperphenylalaninemic ENU1/2 mice

Porous polymer scaffolds surface-modified with arginine-glycine-aspartic acid enhance bone cell attachment and differentiation in vitro

Neuroprotective effects of encapsulated CNTF-producing cells in a rodent model of Huntington's disease are dependent on the proximity of the implant to the lesioned striatum

Reduction Mammaplasty : A Review of Managed Care Medical Policy Coverage Criteria

Sustained release emphasizing recombinant human bone morphogenetic protein-2

Stability of hydrogels used in cell encapsulation: An in vitro comparison of alginate and agarose

Manipulation of Suspended Single Cells by Microfluidics and Optical Tweezers

Gene therapy approaches for modulating bone regeneration

Revascularization of rat fasciocutaneous flap using CROSSEAL® with VEGF protein or plasmid DNA expressing VEGF

Injection of Chemotherapeutic Microspheres and Glioma IV: Eradicating Tumors in Rats

Cell delivery to the central nervous system

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