Guangping Gao

Co-delivered PD-L1 rescues the protective efficacy mediated by an AAV-expressed HIV-1 bNAb

This study shows that co-delivering PD-L1 with an AAV-expressed HIV-1 bNAb reduces anti-drug antibodies and improves bNAb expression and protection against HIV in rhesus macaques.

Overcoming host immune responses to an AAV-delivered HIV-1 bNAb in rhesus macaques mediated by co-delivery of PD-L1

This study shows that co-delivering PD-L1 with AAV-delivered bNAb 10-1074 reduces ADA responses and improves bNAb durability in rhesus macaques.

Characterization of expression elements for an AAV delivered antibody in nonhuman primates when co-delivered with PD-L1

This study evaluates AAV transgene cassettes for expressing 10-1074 antibody in nonhuman primates and mice, showing promoter and WPRE effects on expression levels and immune responses.

Durable protection against SIV challenge by adeno-associated virus delivery of Env-specific antibodies

AAV-delivered broadly neutralizing antibodies provide durable protection against SIV challenge in rhesus macaques, while ADCC-only antibodies do not.

Postnatal gene restoration in succinic semialdehyde dehydrogenase deficiency (SSADHD) reveals phenotype reversibility

This study shows that restoring the ALDH5A1 gene in a mouse model of SSADHD reverses hyperactivity and reduces GHB levels, improving survival.

Seipin mediates Perilipin-1 recruitment to lipid droplets to preserve human adipocyte identity

This study shows seipin is essential for recruiting Perilipin-1 to lipid droplets in human adipocytes, maintaining adipocyte identity and preventing de-differentiation.

A single amino acid variant in the variable region I of AAV capsid confers liver detargeting

The paper identifies an amino acid variant in AAV capsid VR-1 that reduces liver targeting, suggesting its potential for minimizing liver toxicity in gene therapy.

A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models

This paper demonstrates that a compact ABE corrects the PiZ mutation in AATD mouse models, reducing liver disease and improving lung function.

Gene therapies alleviate absence epilepsy associated with Scn2a deficiency in DBA/2J mice

This paper shows that gene therapies can reduce absence seizures in DBA/2J mice with Scn2a deficiency by restoring NaV1.2 or introducing human KV1.1.

AAVone: A Cost-Effective, Single-Plasmid Solution for Efficient AAV Production with Reduced DNA Impurities

AAVone is a single-plasmid system for AAV production that increases yield, reduces DNA impurities, and simplifies the manufacturing process.

Behavioral Efficacy of AAV FOXG1 Gene Replacement Therapy in a Mouse Model of FOXG1 Syndrome

This study shows that AAV FOXG1 gene therapy improves behavioral deficits in a mouse model of FOXG1 syndrome with a Q84P mutation.

Adeno-associated viral delivery of Env-specific antibodies prevents SIV rebound after discontinuing antiretroviral therapy

This study shows that delivering Env-specific antibodies via AAV prevents SIV rebound in macaques after stopping ART.

A Comprehensive Atlas of AAV Tropism in the Mouse

This paper maps the tissue tropism of ten AAV serotypes in mice after systemic delivery, identifying liver preference and unexpected transduction in adrenals, testes, and ovaries.

Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope

This paper engineers Nme2Cas9 base editors to improve activity and targeting scope, enabling correction of MECP2 mutations with minimal off-target effects.

Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

Adenine Base Editing in vivo with a Single Adeno-Associated Virus Vector

This study develops a compact adenine base editor (Nme2-ABE) that can be delivered via a single AAV vector to efficiently edit mouse genomic loci and revert disease mutations in a tyrosinemia model.

Paradoxical activation of SREBP1c and de novo lipogenesis by hepatocyte-selective ACLY depletion in obese mice.

The paper shows that ACLY depletion in obese mice paradoxically increases de novo lipogenesis despite reducing AcCoA levels, linked to increased SREBP1c expression.

Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors

This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.

Start codon disruption with CRISPR/Cas9 prevents murine Fuchs' endothelial corneal dystrophy

This study shows that CRISPR/Cas9 gene editing in adult mice prevents vision loss in a model of Fuchs' endothelial corneal dystrophy by disrupting the start codon of the mutant COL8A2 gene.

Synergistic effect of deoxynucleosides and AAV gene therapy for thymidine kinase 2 deficiency

This study shows that combining AAV gene therapy with deoxynucleoside supplementation improves survival and mitochondrial function in Tk2-deficient mice.

Targeted complement inhibition at synapses prevents microglial synaptic engulfment and synapse loss in demyelinating disease

This study identifies synapse loss and microglial synaptic engulfment in MS models and demonstrates that targeted complement inhibition prevents synapse loss and visual dysfunction.

Conditional, inducible gene silencing in dopamine neurons reveals a sex-specific role for Rit2 GTPase in acute cocaine response and striatal function