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Related Concept Videos

Non-nuclear Inheritance01:29

Non-nuclear Inheritance

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Most DNA resides in the nucleus of a cell. However, some organelles in the cell cytoplasm⁠—such as chloroplasts and mitochondria⁠—also have their own DNA. These organelles replicate their DNA independently of the nuclear DNA of the cell in which they reside. Non-nuclear inheritance describes the inheritance of genes from structures other than the nucleus.
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Inheritance of Chromatin Structures03:17

Inheritance of Chromatin Structures

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Epigenetics is the study of inherited changes in a cell's phenotype without changing the DNA sequences. It provides a form of memory for the differential gene expression pattern to maintain cell lineage, position-effect variegation, dosage compensation, and maintenance of chromatin structures such as telomeres and centromeres. For example, the structure and location of the centromere on chromosomes are epigenetically inherited. Its functionality is not dictated or ensured by the underlying...
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Chromosomal Theory of Inheritance01:39

Chromosomal Theory of Inheritance

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In 1866, Gregor Mendel published the results of his pea plant breeding experiments, providing evidence for predictable patterns in the inheritance of physical characteristics. The significance of his findings was not immediately recognized. In fact, the existence of genes was unknown at the time. Mendel referred to hereditary units as “factors.”
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Genomic Imprinting and Inheritance02:30

Genomic Imprinting and Inheritance

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Diploid organisms inherit genetic material through chromosomes from both parents. Copies of the same gene are known as alleles. In most cases, both alleles are simultaneously expressed and allow various cellular processes to function optimally. If one of the alleles is missing or mutated, the expression of the other allele can compensate; however, this is not true for all genes.
The expression of some genes depends on which parent passed the gene to the offspring, through a phenomenon known as...
37.2K
Inheritance01:25

Inheritance

1.6K
Gregor Mendel's pioneering work on the principles of inheritance fundamentally transformed our understanding of how traits are transmitted from generation to generation. His experiments with pea plants laid the groundwork for the discovery of genes, discrete units within organisms that control heredity.
Each gene exists in pairs, and the combination of these genes from both parents forms an individual's genotype. This genotype is a blueprint of potential traits. Examples of genotype...
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Disorders of Acid-Base Balance01:29

Disorders of Acid-Base Balance

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The human body maintains a precise pH range of arterial blood between 7.35 and 7.45. Deviations result in either acidosis (pH < 7.35) or alkalosis (pH > 7.45). These conditions are further classified as respiratory or metabolic disorders based on their underlying cause.
Respiratory Acidosis and Alkalosis
Respiratory acidosis occurs due to an increase in the partial pressure of carbon dioxide PCO2 in the blood. It often arises from shallow breathing or impaired gas exchange caused by...
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Related Experiment Video

Updated: Feb 3, 2026

High-throughput Screening for Protein-based Inheritance in S. cerevisiae
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RNA-Based Therapies for Inherited Metabolic Disorders.

Reddy Sreekanth Vootukuri1,2, Sonam Gurung1,2, Roopkatha Ghosh1

  • 1Genetics and Genomic Medicine, University College London Great Ormond Street Institute of Child Health, London, UK.

Journal of Inherited Metabolic Disease
|February 1, 2026
PubMed
Summary
This summary is machine-generated.

RNA-based therapies offer new hope for inherited metabolic disorders (IMDs). This review explores RNA chemistry, delivery methods, and clinical applications for these complex genetic conditions.

Keywords:
AONRNA therapygene silencinginborn errors of metabolismmRNAn‐of‐1siRNA

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Area of Science:

  • Genetics and Molecular Biology
  • Pharmacology and Therapeutics

Background:

  • Inherited metabolic disorders (IMDs) are genetic conditions with limited treatment options.
  • RNA-based therapies are emerging as promising interventions for IMDs.

Purpose of the Study:

  • To review RNA-based therapy modalities for IMDs.
  • To discuss challenges and advancements in RNA delivery systems.
  • To highlight clinical applications and future directions.

Main Methods:

  • Review of RNA therapy chemistry and mechanisms (AONs, siRNAs, mRNA).
  • Analysis of delivery methods (LNPs, ligand conjugation, tissue-specific systems).
  • Summary of clinical applications in IMDs and rare diseases.

Main Results:

  • RNA therapies can modulate gene expression, silence transcripts, and restore proteins.
  • Lipid nanoparticles and other methods are advancing RNA delivery.
  • N-of-1 therapies demonstrate personalized medicine potential for IMDs.

Conclusions:

  • RNA-based therapeutics hold significant potential for treating IMDs.
  • Continued innovation in chemistry and delivery is crucial.
  • Streamlined regulatory frameworks are needed to advance RNA therapies for IMDs.