Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects
A Herzeg, B Borges, BR Lianoglou… - Prenatal …, 2023 - Wiley Online Library
Lysosomal storage disorders (LSDs) are a group of monogenic condition, with many
characterized by an enzyme deficiency leading to the accumulation of an undegraded …
characterized by an enzyme deficiency leading to the accumulation of an undegraded …
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice
Angelman syndrome (AS), an early-onset neurodevelopmental disorder characterized by
abnormal gait, intellectual disabilities, and seizures, occurs when the maternal allele of the …
abnormal gait, intellectual disabilities, and seizures, occurs when the maternal allele of the …
Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus
B Borges, A Varthaliti, M Schwab, MT Clarke… - … Therapy Methods & …, 2024 - cell.com
Prenatal somatic cell gene therapy (PSCGT) could potentially treat severe, early-onset
genetic disorders such as spinal muscular atrophy (SMA) or muscular dystrophy. Given the …
genetic disorders such as spinal muscular atrophy (SMA) or muscular dystrophy. Given the …
[图书][B] Exploring and Exploiting Genetic Risk for Psychiatric Disorders
An edited volume that looks at the state of psychiatric genetics and how to chart a path
forward. In this edited collection, experts from psychiatric and statistical genetics …
forward. In this edited collection, experts from psychiatric and statistical genetics …
[HTML][HTML] The neonatal adverse event severity scale: current status, a stakeholders' assessment, and future perspectives
K Allegaert, T Salaets, K Wade, MA Short… - Frontiers in …, 2024 - frontiersin.org
To support informed decisions on drug registration and prescription, clinical trials need tools
to assess the efficacy and safety signals related to a given therapeutic intervention …
to assess the efficacy and safety signals related to a given therapeutic intervention …
Acceptability of prenatal diagnosis and prenatal treatment of haemophilia using cell and gene therapies within US haemophilia community
QDM Pham, SM Thomson, BN Schaible, KD Mills… - …, 2023 - Wiley Online Library
Background The overall burden of disease in persons with haemophilia continues to be high
despite the latest advancements in therapeutics. Clinical trials testing prenatal treatments for …
despite the latest advancements in therapeutics. Clinical trials testing prenatal treatments for …
Prenatal Somatic Cell Gene Therapy
Numerous prenatally diagnosed anatomic conditions have become amenable to intrauterine
procedures due to our ability to capitalize on novel diagnostic tools such as prenatal …
procedures due to our ability to capitalize on novel diagnostic tools such as prenatal …
Concluding Summary
As discussed throughout this volume, our understanding of the genetic basis of psychiatric
disorders has advanced tremendously. This understanding now needs to be translated into …
disorders has advanced tremendously. This understanding now needs to be translated into …