Parkinson's disease: a neurodegenerative disease particularly amenable to gene therapy

Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex.

Gene therapy·2015

Combining glial cell line-derived neurotrophic factor gene delivery (AdGDNF) with L-arginine decreases contusion size but not behavioral deficits after traumatic brain injury.

Brain research·2011

Distribution and morphology of the catecholaminergic neural elements in the human hypothalamus.

Neuroscience·2010

Glial cell line-derived neurotrophic factor gene therapy ameliorates chronic hyperprolactinemia in senile rats.

Neuroscience·2010

Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors.

Gene therapy·2010

Differentiated dopaminergic MN9D cells only partially recapitulate the electrophysiological properties of midbrain dopaminergic neurons.

Developmental neuroscience·2006

Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

A phase 1/2 dose-escalation study of sepiapterin in patients with 6-pyruvoyl-tetrahydropterin synthase deficiency with hyperphenylalaninemia.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

D-Amino Acid Prodrug DLMEH Activates mTORC1 via Sestrin2 to Restore Muscle Protein Synthesis in Sarcopenia.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Combination Of Bispecific Antibodies Enable Targeted TNFRSF Agonism and Effective Antitumor Activity.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Protein-based masks to unlock conditional activation of therapeutics.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Optimization of AsCas12f1-Mediated Long-Term Gene Repression.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Treatment with CFTR modulators and subsequent remission of AA amyloidosis.

Clinical kidney journal·2026

Correction: Targeting oxidized phosphatidylcholines in SOD1-associated ALS: therapeutic potential of PC-OxPL-VecTab®.

Frontiers in neuroscience·2026

Comparison of Peg-INF plus TDF versus TDF Monotherapy in Indeterminate-phase Chronic Hepatitis B Patients with High HBsAg levels, HBeAg-negative status, and Normal ALT Levels.

Journal of clinical and translational hepatology·2026

Novel and Emerging Therapies for Childhood-Onset Movement Disorders.

Movement disorders : official journal of the Movement Disorder Society·2026

Implementation of soluble CD38 in blood crossmatching for patients on anti-CD38 therapy: A single-center experience.

Transfusion·2026

FAP+ pericyte-like cells promote monocyte differentiation into tumor-associated macrophages in glioblastoma.

Experimental & molecular medicine·2026

Related Experiment Videos

Parkinson's disease: a neurodegenerative disease particularly amenable to gene therapy

Related Concept Videos

Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex.

Combining glial cell line-derived neurotrophic factor gene delivery (AdGDNF) with L-arginine decreases contusion size but not behavioral deficits after traumatic brain injury.

Distribution and morphology of the catecholaminergic neural elements in the human hypothalamus.

Glial cell line-derived neurotrophic factor gene therapy ameliorates chronic hyperprolactinemia in senile rats.

Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors.

Differentiated dopaminergic MN9D cells only partially recapitulate the electrophysiological properties of midbrain dopaminergic neurons.

Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.

A phase 1/2 dose-escalation study of sepiapterin in patients with 6-pyruvoyl-tetrahydropterin synthase deficiency with hyperphenylalaninemia.

D-Amino Acid Prodrug DLMEH Activates mTORC1 via Sestrin2 to Restore Muscle Protein Synthesis in Sarcopenia.

Combination Of Bispecific Antibodies Enable Targeted TNFRSF Agonism and Effective Antitumor Activity.

Protein-based masks to unlock conditional activation of therapeutics.

Optimization of AsCas12f1-Mediated Long-Term Gene Repression.

Treatment with CFTR modulators and subsequent remission of AA amyloidosis.

Correction: Targeting oxidized phosphatidylcholines in SOD1-associated ALS: therapeutic potential of PC-OxPL-VecTab®.

Comparison of Peg-INF plus TDF versus TDF Monotherapy in Indeterminate-phase Chronic Hepatitis B Patients with High HBsAg levels, HBeAg-negative status, and Normal ALT Levels.

Novel and Emerging Therapies for Childhood-Onset Movement Disorders.

Implementation of soluble CD38 in blood crossmatching for patients on anti-CD38 therapy: A single-center experience.

FAP+ pericyte-like cells promote monocyte differentiation into tumor-associated macrophages in glioblastoma.

Related Experiment Videos

Parkinson's disease: a neurodegenerative disease particularly amenable to gene therapy

Related Concept Videos

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